prednisone and Muscular Dystrophy, Duchenne studies

Prednisone: A synthetic anti-inflammatory glucocorticoid derived from CORTISONE. It is biologically inert and converted to PREDNISOLONE in the liver.
prednisone : A synthetic glucocorticoid drug that is particularly effective as an immunosuppressant, and affects virtually all of the immune system. Prednisone is a prodrug that is converted by the liver into prednisolone (a beta-hydroxy group instead of the oxo group at position 11), which is the active drug and also a steroid.

Muscular Dystrophy, Duchenne: An X-linked recessive muscle disease caused by an inability to synthesize DYSTROPHIN, which is involved with maintaining the integrity of the sarcolemma. Muscle fibers undergo a process that features degeneration and regeneration. Clinical manifestations include proximal weakness in the first few years of life, pseudohypertrophy, cardiomyopathy (see MYOCARDIAL DISEASES), and an increased incidence of impaired mentation. Becker muscular dystrophy is a closely related condition featuring a later onset of disease (usually adolescence) and a slowly progressive course. (Adams et al., Principles of Neurology, 6th ed, p1415)

Research Excerpts

Excerpt	Relevance	Reference
"Prednisone and deflazacort are steroids that help to reduce muscle inflammation and are used as treatments for DMD."	5.22	Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study. ( Cook, T; Miller, H; Wong, BL, 2022)
" The main outcome measures were prevalence of cataracts and elevated IOP, age of first detection of cataract, time from initial steroid use to first detection of cataract, and relative risk of cataract development for deflazacort versus prednisone treatment."	3.88	Cataract development associated with long-term glucocorticoid therapy in Duchenne muscular dystrophy patients. ( Horn, PS; Rice, ML; Wong, B; Yang, MB, 2018)
"In this single center, open label pilot study, once-weekly prednisone was safe and well tolerated."	3.11	An Open Label Exploratory Clinical Trial Evaluating Safety and Tolerability of Once-Weekly Prednisone in Becker and Limb-Girdle Muscular Dystrophy. ( Ajroud-Driss, S; Casey, P; Joslin, BC; McNally, EM; Zelikovich, AS, 2022)
"Among patients with Duchenne muscular dystrophy, treatment with daily prednisone or daily deflazacort, compared with intermittent prednisone alternating 10 days on and 10 days off, resulted in significant improvement over 3 years in a composite outcome comprising measures of motor function, pulmonary function, and satisfaction with treatment; there was no significant difference between the 2 daily corticosteroid regimens."	3.11	Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. ( Alhaswani, Z; Baranello, G; Barohn, RJ; Bello, L; Brown, MW; Burnette, WB; Bushby, K; Butterfield, RJ; Campbell, C; Chang, T; Childs, AM; Ciafaloni, E; Darras, BT; Eagle, M; Finkel, RS; Flanigan, KM; Gangfuss, A; Griggs, RC; Guglieri, M; Han, JJ; Hart, KA; Henricson, EK; Herr, BE; Horrocks, IA; Howard, JF; Hughes, I; Joyce, N; Kang, PB; King, WM; Kirschner, J; Krzesniak-Swinarska, M; Kumar, A; Kuntz, NL; Maggi, L; Mah, JK; Manzur, A; Martens, WB; McColl, E; McDermott, MP; McDonald, CM; McMillan, HJ; Mongini, TE; Morrison, L; O'Reardon, K; Pegoraro, E; Ricci, F; Roper, H; Schara-Schmidt, U; Shieh, PB; Speed, C; Spinty, S; Statland, JM; Straub, V; Tawil, R; Thangarajh, M; van Ruiten, H; Vita, G; Vita, GL; von der Hagen, M; Wicklund, M; Wilichowski, E; Wilkinson, J; Willis, T, 2022)
" Physician-reported incidences of adverse events (AEs) for Cushingoid appearance, hirsutism, weight gain, and behavior change were less for vamorolone than published incidences for prednisone and deflazacort."	2.94	Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study. ( Arrieta, A; Castro, D; Clemens, PR; Conklin, LS; Damsker, JM; Dang, UJ; Finkel, RS; Gordish-Dressman, H; Guglieri, M; Hagerty, L; Hoffman, EP; Jaros, M; Kerchner, L; Kuntz, NL; Mah, JK; McDonald, CM; Mengle-Gaw, LJ; Morgenroth, LP; Nevo, Y; Ryan, MM; Schwartz, BD; Shale, P; Shimony, M; Smith, EC; Tulinius, M; Webster, R, 2020)
" The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis."	2.82	Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. ( Cwik, V; Dubow, JS; Fehlings, DL; Florence, JM; Greenberg, CR; Griggs, RC; King, W; Kissel, JT; Mendell, JR; Meyer, JM; Miller, JP; Moxley, RT; Pandya, S; Pestronk, A; Vanasse, M, 2016)
" Differences in standards of care and dosing complicate interpretation of this finding, but stratification by PRED/DFZ might be considered in clinical trials."	2.80	Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study. ( Bello, L; Cnaan, A; Duong, T; Gordish-Dressman, H; Henricson, EK; Hoffman, EP; McDonald, CM; Morgenroth, LP, 2015)
"Equivalence was met for weekend and daily dosing of prednisone for the primary outcomes of quantitative muscle testing (QMT) arm score and QMT leg score."	2.76	Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy. ( Arrieta, A; Bertorini, TE; Clemens, PR; Cnaan, A; Connolly, AM; Day, JW; Escolar, DM; Florence, J; Hache, LP; Henricson, E; Hu, F; Kornberg, AJ; Lotze, T; Mayhew, J; McDonald, CM; Monasterio, E; Nevo, Y; Pestronk, A; Ryan, MM; Viswanathan, V; Zimmerman, A, 2011)
"Prednisone treatment is used to prolong ambulation in patients with Duchenne muscular dystrophy (DMD)."	2.71	Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial. ( Beenakker, EA; Brouwer, OF; Fock, JM; Koopman, HM; Maurits, NM; Van der Hoeven, JH; Van Tol, MJ, 2005)
" We used a different dosing regimen of prednisone to determine if benefit to boys with Duchenne muscular dystrophy might be maintained with fewer side effects."	2.70	High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. ( Connolly, AM; Florence, J; Renna, R; Schierbecker, J, 2002)
"We randomized 18 Duchenne muscular dystrophy (DMD) boys whose age ranged from 5."	2.69	A multicenter, double-blind, randomized trial of deflazacort versus prednisone in Duchenne muscular dystrophy. ( Angelini, C; Berardinelli, A; Bonifati, MD; Bonometto, P; Gorni, K; Lanzi, G; Orcesi, S; Ruzza, G, 2000)
"In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials."	2.66	Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials. ( Cox, DA; Darras, BT; Elfring, G; Landry, J; McDonald, CM; McDonnell, E; Peltz, SW; Sajeev, G; Shieh, PB; Signorovitch, J; Souza, M; Yao, Z, 2020)
"Treatment of DMD patients with the corticosteroids prednisone or deflazacort remains the standard of care, and recent data shows that early treatment (as young as 5 months) with a weekend dosing regimen results in measurable improvement in motor outcomes."	2.61	Update in Duchenne and Becker muscular dystrophy. ( Flanigan, KM; Waldrop, MA, 2019)
"We have extended the scope of this updated review to include comparisons of different corticosteroids and dosing regimens."	2.53	Corticosteroids for the treatment of Duchenne muscular dystrophy. ( Brassington, R; Jichi, F; Kuntzer, T; Manzur, AY; Matthews, E, 2016)
" There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen."	2.48	Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy. ( Bushby, K; Connor, EM; Damsker, J; Hoffman, EP; McCall, JM; Nagaraju, K; Reeves, E, 2012)
"Duchenne muscular dystrophy is the most common muscular dystrophy of childhood."	2.42	Glucocorticoid corticosteroids for Duchenne muscular dystrophy. ( Kuntzer, T; Manzur, AY; Pike, M; Swan, A, 2004)
"The most common myopathy in children, Duchenne muscular dystrophy (DMD), is the focus of active pharmacologic clinical trials."	2.41	Pharmacologic and genetic therapy for childhood muscular dystrophies. ( Escolar, DM; Scacheri, CG, 2001)
"Duchenne muscular dystrophy is the most common and most severe form of childhood muscular dystrophies, resulting in early loss of ambulation between the ages of 7 and 13 years and death in the teens and twenties."	2.41	Corticosteroids in Duchenne muscular dystrophy: a reappraisal. ( Christopher, C; Wong, BL, 2002)
"Dimethyl fumarate (DMF) is a potent immunomodulatory small molecule nuclear erythroid 2-related factor 2 activator with current clinical utility in the treatment of multiple sclerosis and psoriasis that could be effective for DMD and rapidly translatable."	1.91	Dimethyl fumarate modulates the dystrophic disease program following short-term treatment. ( Apostolopoulos, V; Arthur, PG; Bagaric, RM; Bautista, AP; Campelj, DG; Dargahi, N; de Haan, JB; Debrincat, D; Debruin, DA; Fischer, D; Guven, N; Hafner, P; Kourakis, S; Pompeani, N; Ritenis, EJ; Rybalka, E; Sahakian, L; Stupka, N; Terrill, JR; Timpani, CA, 2023)
" Glucocorticoids such as prednisone and deflazacort have powerful anti-inflammatory benefits and are the standard of care for DMD, but their long-term use can result in severe adverse side effects; thus, vamorolone was designed as a unique dissociative steroidal anti-inflammatory drug, to retain efficacy and minimise these adverse effects."	1.91	Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy. ( Grounds, MD; Lloyd, EM, 2023)
"Characterize and compare disease progression by steroid treatment (prednisone, deflazacort, or no steroids) among non-ambulatory boys with DMD."	1.91	Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1. ( Frean, M; Freimark, J; Goemans, N; Henricson, EK; Hor, KN; Koladicz, K; Lane, H; Marden, JR; Mayer, OH; McDonald, CM; Miller, D; Signorovitch, J; Trifillis, P; Zhang, A, 2023)
"The multidisciplinary Duchenne muscular dystrophy (DMD) Care Considerations were developed to standardize care and improve outcomes."	1.72	Direct costs of adhering to selected Duchenne muscular dystrophy Care Considerations: Estimates from a midwestern state. ( Conway, KM; Grosse, SD; Ouyang, L; Romitti, PA; Street, N, 2022)
"We evaluated biomarkers from Duchenne Muscular Dystrophy patients, finding that, compared with chronic daily steroid use, weekend steroid use was associated with reduced serum insulin, free fatty acids, and branched chain amino acids, as well as reduction in fat mass despite having similar BMIs."	1.51	Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming. ( Barish, GD; Bass, J; Demonbreun, AR; Haldar, SM; Jiang, Z; Kuntz, NL; McNally, EM; Peek, CB; Quattrocelli, M; Zelikovich, AS, 2019)
"Corticosteroids are highly prescribed and effective anti-inflammatory drugs but the burden of side effects with chronic use significantly detracts from patient quality of life, particularly in children."	1.48	Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. ( Damsker, JM; Fiorillo, AA; Heier, CR; Hoffman, EP; Nagaraju, K; Tully, CB, 2018)
" Although daily glucocorticoid therapy has been shown to extend ambulatory function in DMD, less frequent dosing is often used because of side effect concerns."	1.46	Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment. ( Jefferies, JL; McMahon, MA; Rutter, MM; Rybalsky, I; Sawnani, H; Shellenbarger, KC; Tian, C; Wong, BL, 2017)
" Herein, we assessed the efficacy of steroid dosing on sarcolemmal repair, muscle function, histopathology, and the regenerative capacity of primary muscle cells."	1.46	Intermittent Glucocorticoid Dosing Improves Muscle Repair and Function in Mice with Limb-Girdle Muscular Dystrophy. ( Demonbreun, AR; McNally, EM; Page, PG; Quattrocelli, M; Salamone, IM; Warner, JL, 2017)
" Chronic steroid use usually involves once-daily dosing, although weekly dosing in children has been suggested for its reduced side effects on behavior."	1.46	Intermittent glucocorticoid steroid dosing enhances muscle repair without eliciting muscle atrophy. ( Barefield, DY; Demonbreun, AR; Earley, JU; Hadhazy, M; McNally, EM; Quattrocelli, M; Vo, AH; Warner, JL, 2017)
"Patients with Duchenne muscular dystrophy (DMD) develop dilated cardiomyopathy and are at risk for kidney injury."	1.43	Identifying evidence of cardio-renal syndrome in patients with Duchenne muscular dystrophy using cystatin C. ( Goldstein, SL; Jefferies, JL; Kaddourah, A; Mathew, J; Ryan, TD; Villa, CR; Wong, BL, 2016)
" Growth curve comparisons and linear mixed-effects modeling, adjusted for race/ethnicity and birth year, were used to evaluate growth and steroid treatment patterns (age at initiation, dosing interval, duration, cumulative dose)."	1.43	Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy. ( Ciafaloni, E; DiGuiseppi, C; James, K; Lamb, MM; Ouyang, L; Pandya, S; Weitzenkamp, D; West, NA; Yang, M, 2016)
"Prednisone was the positive control in both studies."	1.42	Chronic Dosing with Membrane Sealant Poloxamer 188 NF Improves Respiratory Dysfunction in Dystrophic Mdx and Mdx/Utrophin-/- Mice. ( Kernodle, S; Markham, BE; Nemzek, J; Sigler, R; Wilkinson, JE, 2015)
"Patients with Duchenne muscular dystrophy exhibit progressive cardiac and skeletal muscle dysfunction."	1.42	Myocardial fibrosis burden predicts left ventricular ejection fraction and is associated with age and steroid treatment duration in duchenne muscular dystrophy. ( Benson, DW; Fleck, RJ; Gao, Z; Hor, KN; Jefferies, JL; Mazur, W; Sticka, JJ; Tandon, A; Taylor, MD; Towbin, JA; Villa, CR; Wong, BL, 2015)
"The prednisone-mediated increase in α7 integrin was associated with increased laminin-α2 in prednisone-treated dystrophin-deficient muscle."	1.39	Levels of α7 integrin and laminin-α2 are increased following prednisone treatment in the mdx mouse and GRMD dog models of Duchenne muscular dystrophy. ( Burkin, DJ; Kornegay, JN; Sarathy, A; Wuebbles, RD, 2013)
"Prednisone treatment delays the progressive course of Duchenne muscular dystrophy."	1.38	Normal height and weight in a series of ambulant Duchenne muscular dystrophy patients using the 10 day on/10 day off prednisone regimen. ( de Groot, IJ; Hendriks, JC; Noordam, C; Sie, LT; ten Dam, K; van Alfen, N, 2012)
"Short stature and delayed puberty were the most relevant side effects."	1.38	Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up. ( Armaroli, A; Cecconi, I; Cicognani, A; Ferlini, A; Franzoni, E; Gennari, M; Gnudi, S; Malaspina, E; Merlini, L; Talim, B, 2012)
"2 years; dosing regimen prednisone 0."	1.38	Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure. ( Collins, J; Cripe, LH; Hor, KN; Rose, SR; Rutter, MM; Sawnani, H; Sucharew, H; Wong, BL; Woo, JG, 2012)
"Prednisone treatment in mdx mice resulted in decreased CTSB activity and increased grip strength in forelimbs and hindlimbs."	1.37	Non-invasive optical imaging of muscle pathology in mdx mice using cathepsin caged near-infrared imaging. ( Baudy, AR; Hoffman, EP; Johnston, HK; Jordan, S; Kesari, A; Nagaraju, K; Sali, A, 2011)
"Arginine butyrate treatment improved grip strength and decreased fibrosis in the gastrocnemius muscle, but did not produce significant improvement in muscle and cardiac histology, heart function, behavioral measurements, or serum creatine kinase levels."	1.36	Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular Dystrophy. ( Becker, M; Bossi, L; Cha, HJ; de la Porte, S; Escolar, DM; Farajian, V; Francia, D; Gernapudi, R; Gordish-Dressman, H; Guerron, AD; Hoffman, EP; Nagaraju, K; Pandey, GS; Partridge, T; Pistilli, E; Rawat, R; Sali, A; Spurney, CF; Zerr, P, 2010)
"Prednisone was started during the ambulant phase at age 3."	1.35	Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy. ( de Groot, IJ; Overweg-Plandsoen, WC; Straathof, CS; van den Burg, GJ; van der Kooi, AJ; Verschuuren, JJ, 2009)
" Two class I trials examined the effect of lower dosage of prednisone (0."	1.33	Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. ( Ashwal, S; Baumbach, L; Connolly, A; Florence, J; Mathews, K; McDonald, C; Moxley, RT; Pandya, S; Sussman, M; Wade, C, 2005)
"Their usage in Duchenne muscular dystrophy may prolong ambulation and upper limb function with similar potency."	1.33	Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect. ( Balaban, B; Carry, T; Clayton, GH; Matthews, DJ, 2005)
" The recommended dosage is 0."	1.33	[Guideline on the use of corticosteroids in Duchenne muscular dystrophy from paediatric neurologists, neurologists and rehabilitation physicians]. ( de Groot, IJ, 2006)
"Steroid administration is beneficial in Duchenne muscular dystrophy (DMD), but the response, incidence, and the severity of side effects are variable."	1.33	The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy. ( Angelini, C; Bonifati, DM; Ermani, M; Hoffman, EP; Pegoraro, E; Witchel, SF, 2006)
"Prednisone therapy was associated with: (i) functional improvement of overall motor disability, in upper limbs of DMD (P < 0."	1.33	The effects of glucocorticoid therapy on the inflammatory and dendritic cells in muscular dystrophies. ( Abu-Dief, EE; Hamed, SA; Hussein, MR; Kamel, NF; Kandil, MR; Mostafa, MG, 2006)
"Duchenne muscular dystrophy is a degenerative muscular disease caused by mutations in the dystrophin gene."	1.32	Prednisone reduces muscle degeneration in dystrophin-deficient Caenorhabditis elegans. ( Carre-Pierrat, M; Gaud, A; Ségalat, L; Simon, JM; Wermuth, CG; Witzel, T, 2004)
"Two boys with Becker's muscular dystrophy had a dramatic and sustained improvement in strength with therapeutic use of pred nisone."	1.31	Prednisone therapy in Becker's muscular dystrophy. ( Johnsen, SD, 2001)

Research

Studies (94)

Authors

Clinical Trials (15)

Trial Overview

Trial Outcomes

6 Minute Walk Test

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	28 (29.79)	29.6817
2010's	45 (47.87)	24.3611
2020's	21 (22.34)	2.80

Authors	Studies
Kaviani, A	1
Khansari Nejad, N	1
Akour, A	1
AlMuhaissen, SA	1
Nusair, MB	1
Al-Tammemi, AB	1
Mahmoud, NN	1
Jalouqa, S	1
Alrawashdeh, MN	1
Shanahan, JP	1
Moore, CM	1
Kampf, JW	1
Szymczak, NK	1
Guo, J	1
Wang, H	1
Luo, Y	1
An, H	1
Zhang, Z	2
Liu, G	1
Li, J	1
Metcalfe, GD	1
Smith, TW	1
Hippler, M	1
Ayala Izurieta, JE	1
Márquez, CO	1
García, VJ	1
Jara Santillán, CA	1
Sisti, JM	1
Pasqualotto, N	1
Van Wittenberghe, S	1
Delegido, J	1
Kubo, Y	1
Kitagawa, Y	1
Miyazaki, T	1
Sohda, M	1
Yamaji, T	1
Sakai, M	1
Saeki, H	1
Nemoto, K	1
Oyama, T	2
Muto, M	1
Takeuchi, H	1
Toh, Y	1
Matsubara, H	1
Mano, M	1
Kono, K	1
Kato, K	1
Yoshida, M	1
Kawakubo, H	1
Booka, E	1
Yamatsuji, T	1
Kato, H	1
Ito, Y	1
Ishikawa, H	1
Ishihara, R	1
Tsushima, T	1
Kawachi, H	1
Kojima, T	1
Kuribayashi, S	1
Makino, T	1
Matsuda, S	1
Doki, Y	1
Ma, P	1
Tang, X	1
Zhang, L	2
Wang, X	1
Wang, W	2
Zhang, X	1
Wang, S	2
Zhou, N	1
Dodd, J	1
Jordan, R	1
Makhlina, M	1
Pesco Koplowitz, L	1
Koplowitz, B	1
Barnett, K	1
Yang, WH	1
Spana, C	1
Buchanan, SW	1
Mafa-Attoye, T	1
Dunfield, K	1
Thevathasan, NV	1
Isaac, ME	1
Wu, X	2
Huang, JW	1
Su, BK	1
Yuan, L	1
Zheng, WQ	1
Zhang, H	2
Zheng, YX	1
Zhu, W	1
Chou, PT	1
Manso, AP	1
De Morais, DC	1
Yamamoto, K	1
Owen, G	1
de Carvalho, RM	1
Palma-Dibb, RG	1
Brown, JC	1
Yang, S	1
Mire, EF	1
Miele, L	1
Ochoa, A	1
Zabaleta, J	1
Katzmarzyk, PT	1
Moi, CT	1
Bhowmick, S	1
Qureshi, M	1
Wang, K	1
Feng, B	1
Yang, Y	1
Chen, Y	1
Wang, Y	3
Yang, L	1
Jiang, K	1
James, TD	1
Chaturvedi, V	1
Postiglione, WM	1
Chakraborty, RD	1
Yu, B	1
Tabiś, W	1
Hameed, S	1
Biniskos, N	1
Jacobson, A	1
Zhou, H	1
Greven, M	1
Ferry, VE	1
Leighton, C	1
Wu, F	1
Lei, H	1
Chen, G	1
Chen, C	1
Song, Y	1
Cao, Z	1
Zhang, C	2
Zhou, J	1
Lu, Y	1
Pappone, C	1
Santinelli, V	1
Mecarocci, V	1
Tondi, L	1
Ciconte, G	1
Manguso, F	1
Sturla, F	1
Vicedomini, G	1
Micaglio, E	1
Anastasia, L	1
Pica, S	1
Camporeale, A	1
Lombardi, M	1
Shieh, PB	6
Elfring, G	4
Trifillis, P	4
Santos, C	1
Peltz, SW	3
Parsons, JA	1
Apkon, S	1
Darras, BT	5
Campbell, C	4
McDonald, CM	13
Galaviz, KI	1
Shah, NS	1
Gutierrez, M	1
Collins, LF	1
Lahiri, CD	1
Moran, CA	1
Szabo, B	1
Sumitani, J	1
Rhodes, J	1
Marconi, VC	1
Nguyen, ML	1
Cantos, VD	1
Armstrong, WS	1
Colasanti, JA	1
Conway, KM	1
Grosse, SD	1
Ouyang, L	2
Street, N	1
Romitti, PA	1
Zelikovich, AS	2
Joslin, BC	1
Casey, P	1
McNally, EM	5
Ajroud-Driss, S	1
Guglieri, M	5
Bushby, K	4
McDermott, MP	2
Hart, KA	2
Tawil, R	2
Martens, WB	2
Herr, BE	2
McColl, E	2
Speed, C	1
Wilkinson, J	2
Kirschner, J	3
King, WM	2
Eagle, M	2
Brown, MW	2
Willis, T	2
Griggs, RC	3
Straub, V	3
van Ruiten, H	1
Childs, AM	3
Ciafaloni, E	4
Spinty, S	3
Maggi, L	1
Baranello, G	3
Butterfield, RJ	2
Horrocks, IA	1
Roper, H	2
Alhaswani, Z	1
Flanigan, KM	5
Kuntz, NL	6
Manzur, A	1
Kang, PB	1
Morrison, L	2
Krzesniak-Swinarska, M	1
Mah, JK	5
Mongini, TE	1
Ricci, F	1
von der Hagen, M	2
Finkel, RS	5
O'Reardon, K	1
Wicklund, M	2
Kumar, A	1
Han, JJ	1
Joyce, N	1
Henricson, EK	3
Schara-Schmidt, U	1
Gangfuss, A	1
Wilichowski, E	2
Barohn, RJ	2
Statland, JM	1
Vita, G	3
Vita, GL	1
Howard, JF	2
Hughes, I	2
McMillan, HJ	3
Pegoraro, E	4
Bello, L	3
Burnette, WB	1
Thangarajh, M	2
Chang, T	1
Asma, A	1
Ulusaloglu, AC	1
Shrader, MW	1
Mackenzie, WG	1
Heinle, R	1
Scavina, M	1
Howard, JJ	1
Wong, BL	9
Cook, T	1
Miller, H	1
Biggar, WD	1
Skalsky, A	1
Clemens, PR	4
Perlman, SJ	1
Smith, EC	3
Horrocks, I	2
Deconinck, N	1
Goemans, N	3
Haberlova, J	1
Mengle-Gaw, LJ	3
Schwartz, BD	3
Harper, AD	1
De Waele, L	1
Castro, D	3
Yang, ML	1
Ryan, MM	4
Tulinius, M	3
Webster, R	3
Rao, VK	1
Sbrocchi, AM	1
Selby, KA	1
Monduy, M	1
Nevo, Y	4
Vilchez-Padilla, JJ	1
Nascimento-Osorio, A	1
Niks, EH	1
de Groot, IJM	1
Katsalouli, M	1
James, MK	1
van den Anker, J	2
Damsker, JM	4
Ahmet, A	1
Ward, LM	1
Jaros, M	3
Shale, P	3
Dang, UJ	2
Hoffman, EP	10
Mayer, OH	2
Hor, KN	3
Miller, D	1
Marden, JR	2
Freimark, J	2
Lane, H	1
Zhang, A	1
Frean, M	1
Koladicz, K	1
Signorovitch, J	3
Fischer, R	1
Porter, K	1
Donovan, JM	1
Scavina, MT	1
Armstrong, N	1
Denger, B	1
Hasham, S	1
Peay, H	1
Marullo, AL	1
O'Halloran, KD	1
Timpani, CA	1
Kourakis, S	1
Debruin, DA	1
Campelj, DG	1
Pompeani, N	1
Dargahi, N	1
Bautista, AP	1
Bagaric, RM	1
Ritenis, EJ	1
Sahakian, L	1
Debrincat, D	1
Stupka, N	1
Hafner, P	1
Arthur, PG	1
Terrill, JR	1
Apostolopoulos, V	1
de Haan, JB	1
Guven, N	1
Fischer, D	1
Rybalka, E	1
Grounds, MD	1
Lloyd, EM	1
Smith, AL	1
Morgenroth, LP	3
Arrieta, A	3
Shimony, M	2
Siener, C	1
McCall, JM	2
Nagaraju, K	6
Conklin, LS	2
Cnaan, A	3
Gordish-Dressman, H	4
Sajeev, G	1
Yao, Z	2
McDonnell, E	1
Souza, M	2
Cox, DA	1
Landry, J	1
Quattrocelli, M	3
Jiang, Z	1
Peek, CB	1
Demonbreun, AR	3
Barish, GD	1
Haldar, SM	1
Bass, J	1
Tian, C	3
Takeuchi, F	1
Nakamura, H	1
Yonemoto, N	1
Komaki, H	1
Rosales, RL	1
Kornberg, AJ	2
Bretag, AH	1
Dejthevaporn, C	1
Goh, KJ	1
Jong, YJ	1
Kim, DS	1
Khadilkar, SV	1
Shen, D	1
Wong, KT	1
Chai, J	1
Chan, SH	1
Khan, S	1
Ohnmar, O	1
Nishino, I	1
Takeda, S	1
Nonaka, I	1
Agboola, F	1
Lin, GA	1
Fluetsch, N	1
Walton, SM	1
Rind, DM	1
Pearson, SD	1
Tomazoni, SS	1
Casalechi, HL	1
Ferreira, CSB	1
Serra, AJ	1
Dellê, H	1
Brito, RBO	1
de Melo, BL	1
Vanin, AA	1
Ribeiro, NF	1
Pereira, AL	1
Monteiro, KKDS	1
Marcos, RL	1
de Carvalho, PTC	1
Frigo, L	1
Leal-Junior, ECP	1
Kerchner, L	1
Hagerty, L	1
Merlini, L	3
Cecconi, I	2
Parmeggiani, A	1
Cordelli, DM	1
Dormi, A	1
Wei, LJ	1
Muntoni, F	1
Hirtz, D	1
Manzur, AY	4
Schara, U	1
Mongini, T	2
Bryan Burnette, W	1
Barefield, DY	1
Warner, JL	2
Vo, AH	1
Hadhazy, M	1
Earley, JU	1
Salamone, IM	1
Page, PG	1
Yoon, SH	1
Sugamori, KS	1
Grynpas, MD	1
Mitchell, J	1
Rice, ML	1
Wong, B	5
Horn, PS	2
Yang, MB	1
Fiorillo, AA	1
Tully, CB	1
Heier, CR	1
Mcintosh, J	1
Jin, F	1
Narayanan, S	1
Abutaleb, ARA	1
Khan, SS	1
Anderson, AS	1
Carr, JC	1
Wilcox, JE	1
Sawnani, H	3
Darmahkasih, A	1
Rybalsky, I	2
Shellenbarger, KC	2
Rutter, MM	3
Simakajornboon, N	1
Amin, R	2
Gurbani, N	1
Pascoe, J	1
Burrows, C	1
Khirani, S	1
Amaddeo, A	1
Fauroux, B	1
Waldrop, MA	1
Wuebbles, RD	1
Sarathy, A	1
Kornegay, JN	1
Burkin, DJ	1
Campbell, K	2
Viollet, L	1
Gomez, AM	1
Walker, CM	1
Mendell, JR	2
Li, M	1
Cai, Y	1
Zhong, M	1
Zou, L	2
Gong, C	1
Dadgar, S	1
Wang, Z	1
Johnston, H	1
Kesari, A	2
Chen, YW	1
Hill, DA	1
Partridge, TA	1
Giri, M	1
Freishtat, RJ	1
Nazarian, J	1
Xuan, J	1
Kim, S	1
Campbell, KA	1
Fox, DJ	2
Matthews, DJ	2
Valdez, R	1
Hu, J	1
Ye, Y	1
Kong, M	1
Hong, S	1
Cheng, L	1
Wang, Q	1
Qin, J	1
Jiang, L	1
Tandon, A	1
Villa, CR	2
Jefferies, JL	3
Gao, Z	1
Towbin, JA	1
Mazur, W	1
Fleck, RJ	1
Sticka, JJ	1
Benson, DW	1
Taylor, MD	1

Trial	Phase	Enrollment	Study Type	Start Date	Status
Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen[NCT01603407]	Phase 3	196 participants (Actual)	Interventional	2013-01-31	Completed
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)[NCT03439670]	Phase 2	121 participants (Actual)	Interventional	2018-06-29	Completed
A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)[NCT05185622]	Phase 2	54 participants (Anticipated)	Interventional	2022-03-21	Recruiting
A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy[NCT05166109]	Phase 2	39 participants (Anticipated)	Interventional	2022-07-07	Recruiting
Evaluating the Impact of Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)[NCT03319030]		43 participants (Actual)	Observational	2017-09-01	Completed
A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)[NCT03038399]	Phase 2	46 participants (Actual)	Interventional	2017-02-02	Completed
A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)[NCT02760277]	Phase 2	48 participants (Actual)	Interventional	2016-07-28	Completed
A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)[NCT02760264]	Phase 2	48 participants (Actual)	Interventional	2016-06-30	Completed
A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy[NCT01826487]	Phase 3	230 participants (Actual)	Interventional	2013-03-26	Completed
Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy[NCT04054375]	Phase 2	20 participants (Actual)	Interventional	2019-07-01	Completed
Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy[NCT02167217]	Phase 2	25 participants (Actual)	Interventional	2014-04-17	Completed
Low-level Mechanical Vibration, Bone Density, Bone Resorption and Muscular Strength in Ambulant Children Affected by Duchenne Muscular Dystrophy[NCT05281120]		20 participants (Actual)	Interventional	2006-11-30	Completed
Phase I/IIa Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2[NCT03333590]	Phase 1/Phase 2	2 participants (Actual)	Interventional	2017-11-06	Active, not recruiting
A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD[NCT03777319]	Phase 1	2 participants (Actual)	Interventional	2018-12-05	Terminated (stopped due to Inability to recruit participants.)
A Comparative Study of Strategies for Management of Duchenne Myopathy in Assiut University Children Hospital[NCT03633565]	Phase 4	45 participants (Anticipated)	Interventional	2018-09-30	Not yet recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Measures the total distance walked in 6 minutes averaged over all post-baseline follow-up visits through Month 36. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Forced Vital Capacity

Intervention	meters (Least Squares Mean)
Daily Prednisone	384.95
Daily Deflazacort	384.17
Intermittent Prednisone	346.81

Forced vital capacity was measured during a spirometry test. Forced expiratory volume (FEV) measures how much air a person can exhale during a forced breath. Forced vital capacity (FVC) is the total amount of air exhaled during the FEV test. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Fractional Shortening Percent

Intervention	liters (Least Squares Mean)
Daily Prednisone	1.4
Daily Deflazacort	1.4
Intermittent Prednisone	1.5

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Heart Rate

Intervention	percentage of fractional shortening (Least Squares Mean)
Daily Prednisone	33.74
Daily Deflazacort	34.01
Intermittent Prednisone	34.33

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Left Ventricular Ejection Fraction Percent

Intervention	bpm (Least Squares Mean)
Daily Prednisone	94.10
Daily Deflazacort	93.52
Intermittent Prednisone	91.65

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

North Star Ambulatory Assessment (NSAA) Score

Intervention	percentage of ejection fraction (Least Squares Mean)
Daily Prednisone	61.88
Daily Deflazacort	62.65
Intermittent Prednisone	62.45

"The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects.~The activities are graded as follows:~2 - Normal - no obvious modification of activity~1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function." (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Number of Participants Who Tolerated the Regimen

Intervention	score on a scale (Least Squares Mean)
Daily Prednisone	23.7
Daily Deflazacort	24.0
Intermittent Prednisone	20.7

The number of participants who completed 36 months of follow-up on the originally assigned dosage (for weight) of study medication. (NCT01603407)
Timeframe: 3 years

Participant Body Mass Index

Participant Height

Participant Weight

PR Interval

Intervention	Participants (Count of Participants)
Daily Prednisone	36
Daily Deflazacort	36
Intermittent Prednisone	37

Intervention	kilograms/square meter (Least Squares Mean)
Daily Prednisone	18.9
Daily Deflazacort	18.3
Intermittent Prednisone	18.1

Intervention	centimeters (Least Squares Mean)
Daily Prednisone	116.8
Daily Deflazacort	115.3
Intermittent Prednisone	119.9

Intervention	kilograms (Least Squares Mean)
Daily Prednisone	26.3
Daily Deflazacort	24.9
Intermittent Prednisone	26.3

Measured by trans-thoracic echocardiogram and 12-lead ECG. (NCT01603407)
Timeframe: 36 months

Quality of Life - Parent

Intervention	milliseconds (Least Squares Mean)
Daily Prednisone	115.59
Daily Deflazacort	116.87
Intermittent Prednisone	117.90

Quality of life was measured by parent/guardian self-report for all children utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life for the child. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Quality of Life- Child

Intervention	score on a scale (Least Squares Mean)
Daily Prednisone	64.88
Daily Deflazacort	63.71
Intermittent Prednisone	61.33

Quality of life was measured by child self-report in children age 5 and older utilizing the PEDSQL measurement tool. This is a 23-question tool. Scores can range from 0 to 100, with higher scores indicating better quality of life. (NCT01603407)
Timeframe: Average of Months 12, 24, and 36 visits

Range of Motion (Goniometry) of Left Ankle

Intervention	score on a scale (Least Squares Mean)
Daily Prednisone	67.39
Daily Deflazacort	64.96
Intermittent Prednisone	65.07

Range of motion at the ankle joint in dorsiflexion measured in degrees from plantigrade averaged over all post-baseline visits. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Range of Motion (Goniometry) of Right Ankle

Intervention	degrees (Mean)
Daily Prednisone	4.39
Daily Deflazacort	3.29
Intermittent Prednisone	2.67

Rise From the Floor Velocity

Intervention	degrees (Mean)
Daily Prednisone	4.05
Daily Deflazacort	2.81
Intermittent Prednisone	2.29

Reciprocal of time to rise from the floor (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Treatment Satisfaction Questionnaire for Medication (TSQM) Global Satisfaction With Treatment Score

Intervention	rise/sec (Least Squares Mean)
Daily Prednisone	0.24
Daily Deflazacort	0.24
Intermittent Prednisone	0.18

The TSQM Global Satisfaction with Treatment is a 14-item questionnaire that ranges from 0 - 100 with higher scores indicating better outcomes. (NCT01603407)
Timeframe: Average of Months 3, 6, 12, 18, 24, 30 and 36 visits

Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline

Intervention	score on a scale (Least Squares Mean)
Daily Prednisone	71.2
Daily Deflazacort	67.8
Intermittent Prednisone	65.1

Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline to the Week 24 assessment (NCT03439670)
Timeframe: 24 weeks

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE Version 4.03

Intervention	Rises/Seconds (Mean)
Treatment Group 1	-.007
Treatment Group 2	0.054

To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- month Treatment Period, in boys ages 4-7 years with DMD; Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination); (NCT03038399)
Timeframe: 24 months

Total Number of Adverse Events as Assessed by CTCAE Version 4.03

Intervention	Participants (Count of Participants)
Dose Level Group 1	4
Dose Level Group 2	14
Dose Level Group 3	29
Dose Level Group 4	1
Dose Level Group 5	39

To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24-month Treatment Period, in boys ages 4-7 years with DMD. Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). (NCT03038399)
Timeframe: 24 Months

BMI Z-score

Intervention	Events (Number)
	Total Number of AEs	Total Number of TEAEs
Dose Level Group 1	15	14
Dose Level Group 2	34	34
Dose Level Group 3	203	202
Dose Level Group 4	5	5
Dose Level Group 5	302	300

"Summary of BMI Z-score of Safety Population.~Please note 0 is the mean. A negative result indicates a response that is many standard deviations below the mean, and a positive result indicates a response that is many standard deviations above the mean. In this case, the closer the group mean BMI Z-score is to 0 is more favorable." (NCT02760277)
Timeframe: 002 Baseline, 003 Week 12, Week 24

Muscle Function Measured by Time to Stand Test (TTSTAND)- Velocity

Intervention	z score (Mean)
	002 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	1.165	1.103	-0.062	1.004	-0.161
Dose Level Group 2	0.703	0.494	-0.209	0.493	-0.210
Dose Level Group 3	1.200	1.261	0.062	1.242	0.043
Dose Level Group 4	0.695	1.011	0.174	1.330	0.493

To compare the efficacy, as measured by the Time to Stand Test (TTSTAND), of vamorolone administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. untreated DMD historical controls in boys ages 4-7 years with DMD (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by North Star Ambulatory Assessment (NSAA)

Intervention	Rises/Second (Mean)
	002 Baseline	003 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	0.18	0.15	0.18	-0.01	0.18	-0.01
Dose Level Group 2	0.24	0.22	0.23	0.00	0.24	0.00
Dose Level Group 3	0.22	0.24	0.24	0.02	0.26	0.05
Dose Level Group 4	0.19	0.22	0.22	0.02	0.24	0.04

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by North Star Ambulatory Assessment (NSAA) in boys ages 4-7 years with DMD. ***Total NSAA score is being reported. The score can range from 0 to 32. Higher scores (approaching 32) indicate a better outcome assessing functional mobility. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Climb Test (TTCLIMB)- Velocity

Intervention	scores on a scale (Mean)
	002 Baseline	003 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	19.0	20.1	19.3	0.3	19.8	0.8
Dose Level Group 2	20.5	20.8	21.2	0.7	21.6	1.1
Dose Level Group 3	20.0	21.7	21.0	1.0	22.3	2.3
Dose Level Group 4	19.7	20.4	20.4	0.5	22.3	2.5

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Climb Test (TTCLIMB) in boys ages 4-7 years with DMD. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Muscle Strength, Mobility, and Functional Exercise Capacity as Measured by Time to Run/Walk 10 Meters Test (TTRW)- Velocity

Intervention	tasks/ second (Mean)
	002 Baseline	003 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	0.20	0.20	0.21	0.01	0.20	0.00
Dose Level Group 2	0.29	0.29	0.34	0.05	0.30	0.01
Dose Level Group 3	0.29	0.31	0.31	0.02	0.34	0.04
Dose Level Group 4	0.24	0.25	0.26	0.02	0.29	0.05

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by Time to Run/Walk Test (TTRW) in boys ages 4-7 years with DMD. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Muscle Strength, Mobility, and Functional Exercise Capacity vs. Historical Controls as Measured by 6-minute Walk Test (6MWT) Meters

Intervention	meters/ second (Mean)
	002 Baseline	003 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	1.60	1.57	1.54	-0.06	1.55	-0.05
Dose Level Group 2	1.77	1.78	1.77	0.00	1.84	0.06
Dose Level Group 3	1.84	1.86	1.97	0.13	1.90	0.06
Dose Level Group 4	1.64	1.72	1.88	0.26	1.89	0.27

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period, on muscle strength, mobility and functional exercise capacity vs. historical controls as measured by 6-minute Walk Test (6MWT) in boys ages 4-7 years with DMD. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 12, 003 Week 24 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Number of Participants With Adverse Events as Assessed by CTCAE Version 4.03

Intervention	Meters (Mean)
	002 Baseline	003 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	316.2	294.3	312.9	6.0	306.2	-11.6
Dose Level Group 2	331.5	332.2	358.7	20.8	350.4	18.9
Dose Level Group 3	353.9	341.1	393.7	39.8	383.1	29.2
Dose Level Group 4	336.8	335.1	369.9	27.6	372.6	43.9

Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug; To evaluate the long-term safety and tolerability of vamorolone, administered orally at daily doses up to 6.0 mg/kg/day over a 24- week Treatment Period, in boys ages 4-7 years with DMD. (NCT02760277)
Timeframe: 24 weeks

Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH

Intervention	participants (Number)
	Subjects with Any TEAE	Subjects with Any Drug Related TEAE	Subjects with Any CTCAE Grade 3 or Higher TEAE	Discontinuation of Study Drug due to TEAE	Subjects with Any Serious TEAE	Death
Dose Level Group 1	10	1	0	0	0	0
Dose Level Group 2	10	2	0	0	1	0
Dose Level Group 3	11	4	0	0	0	0
Dose Level Group 4	11	5	2	0	2	0

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, 003 Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Serum Pharmacodynamics Biomarkers Measured by Levels of ACTH

Intervention	pg/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline	003 Week 26-29	003 Week 26-29 Change from 002 Baseline
Dose Level Group 1	18.3	15.9	13.0	-5.3	12.2	-6.2	19.8	0.6	9.5	-7.8

Serum Pharmacodynamics Biomarkers Measured by Levels of CTX

Intervention	pg/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 2	18.0	18.6	7.1	-10.5	9.0	-9.1	14.0	-4.0
Dose Level Group 3	21.1	18.2	7.8	-13.3	9.0	-12.0	15.7	-5.4
Dose Level Group 4	19.3	18.4	6.5	-13.5	9.0	-12.2	11.3	-6.3

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Serum Pharmacodynamics Biomarkers Measured by Levels of CTX

Intervention	pg/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline	003 Week 26-29	003 Week 26-29 Change from 002 Baseline
Dose Level Group 1	871.0	915.9	897.1	26.1	885.4	-2.6	1109.3	212.3	1059.3	569.5

To investigate the effects of vamorolone, administered orally at daily doses up to 6.0 mg/kg over a 24-week Treatment Period vs. prednisone-treated historical controls, on serum pharmacodynamic (PD) biomarkers of safety (insulin resistance, adrenal axis suppression, and bone turnover). SomaScan aptamer panels testing 1,200 serum proteins were used to discover a candidate set of prednisone-responsive biomarkers, with a subset of these validating in a longitudinal sample set (individual DMD patients pre/post steroid treatment). These PD biomarkers were assigned to a safety panel or efficacy panel based on comparison to normal controls and information concerning the function of each protein. (NCT02760277)
Timeframe: 002 Baseline, 003 Baseline, 003 Week 8, 003 Week 16, Week 24, 003 Week 26-29 (Note: 002 Baseline is from VBP15-002 4 week study (NCT02760264), previous to VBP15-003)

Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Glucose

Intervention	pg/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 2	935.8	964.4	933.3	-31.6	912.8	-46.3	1235.6	295.6
Dose Level Group 3	936.8	949.8	928.3	-8.5	939.8	3.0	1248.7	346.5
Dose Level Group 4	889.3	989.2	825.5	-59.6	953.7	102.3	1237.0	321.4

Serum Pharmacodynamics Biomarkers Measured by Levels of Fasting Insulin

Intervention	mg/dL (Mean)
	002 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	87.5	81.5	-6.8	80.8	-6.3
Dose Level Group 2	88.9	81.7	-7.6	80.8	-9.0
Dose Level Group 3	89.3	84.3	-5.1	81.3	-8.1
Dose Level Group 4	92.3	86.5	-5.2	84.6	-7.8

Serum Pharmacodynamics Biomarkers Measured by Levels of HbA1c

Intervention	uIU/mL (Mean)
	002 Baseline	003 Week 12	003 Week 12 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 1	5.54	4.17	-1.13	4.23	-1.67
Dose Level Group 2	3.09	2.97	-0.14	3.12	0.34
Dose Level Group 3	3.40	3.89	0.49	4.82	1.36
Dose Level Group 4	3.96	6.97	2.97	7.21	3.26

Intervention	% change (Mean)
	003 Week 8 % Change from 002 Baseline	003 Week 16 % Change from 002 Baseline	003 Week 24 % Change from 002 Baseline
Dose Level Group 2	2.28	2.72	0.08
Dose Level Group 3	1.79	1.30	-1.27
Dose Level Group 4	0.02	1.03	-0.33

Intervention	% change (Mean)
	003 Week 8 % Change from 002 Baseline	003 Week 16 % Change from 002 Baseline	003 Week 24 % Change from 002 Baseline	003 Week 26-29 % Change from 002 Baseline
Dose Level Group 1	0.06	1.70	-1.89	-1.25

Intervention	% of HbA1c (Mean)
	002 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 2	5.22	5.33	0.12	5.35	0.14	5.22	0.00
Dose Level Group 3	5.19	5.28	0.09	5.26	0.07	5.13	-0.07
Dose Level Group 4	5.23	5.25	0.00	5.31	0.05	5.24	-0.02

Serum Pharmacodynamics Biomarkers Measured by Levels of Osteocalcin

Intervention	% of HbA1c (Mean)
	002 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline	003 Week 26-29	003 Week 26-29 Change from 002 Baseline
Dose Level Group 1	5.18	5.18	0.00	5.26	0.08	5.15	-0.10	5.07	-0.07

Intervention	ng/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline	003 Week 26-29	003 Week 26-29 Change from 002 Baseline
Dose Level Group 1	37.94	39.20	36.21	-1.60	39.01	1.07	38.80	-1.34	40.10	-1.23

Serum Pharmacodynamics Biomarkers Measured by Levels of P1NP

Intervention	ng/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 2	35.66	41.84	41.78	6.13	42.23	6.57	51.41	15.75
Dose Level Group 3	41.17	47.91	44.45	3.28	44.60	3.43	51.98	10.81
Dose Level Group 4	44.36	42.81	41.55	-2.01	39.39	-4.17	49.08	5.29

Intervention	ng/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline	003 Week 26-29	003 Week 26-29 Change from 002 Baseline
Dose Level Group 1	555.8	573.9	511.6	-20.3	481.9	-73.8	457.1	-30.8	619.0	-152.0

Total Number of Adverse Events as Assessed by CTCAE Version 4.03

Intervention	ng/mL (Mean)
	002 Baseline	003 Baseline	003 Week 8	003 Week 8 Change from 002 Baseline	003 Week 16	003 Week 16 Change from 002 Baseline	003 Week 24	003 Week 24 Change from 002 Baseline
Dose Level Group 2	480.7	489.3	459.8	-22.9	431.8	-42.4	471.1	2.1
Dose Level Group 3	508.2	492.0	485.2	-23.0	455.7	-52.5	565.5	57.3
Dose Level Group 4	511.5	566.3	402.7	-105.6	488.5	-19.8	526.2	8.7

Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol

Intervention	Events (Number)
	Total Number of AEs	Total Number of TEAEs
Dose Level Group 1	48	48
Dose Level Group 2	44	44
Dose Level Group 3	54	54
Dose Level Group 4	73	72

Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-< 7 years with DMD. (NCT02760264)
Timeframe: Week 2 (pre-dose)

Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose

Intervention	mcg/dL (Mean)
Dose Level Group 1	10.425
Dose Level Group 2	9.755
Dose Level Group 3	7.321
Dose Level Group 4	3.010

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin

Intervention	Week 2 % change from Baseline (Mean)
Dose Level Group 1	-1.8
Dose Level Group 2	-4.2
Dose Level Group 3	0.8
Dose Level Group 4	-1.2

Metabolites in Safety Testing (MIST) Assessment

Intervention	Week 2 % change from Baseline (Mean)
Dose Level Group 1	-5.54
Dose Level Group 2	26.07
Dose Level Group 3	42.85
Dose Level Group 4	83.55

A portion of each blood sample of the Week 2 (Day 14) pharmacokinetic assessment time points for the subjects receiving vamorolone 2 mg/kg/day was used for analysis of vamorolone metabolites. (NCT02760264)
Timeframe: Week 2 (Day 14)

Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03

Intervention	% of total drug related exposure (Mean)
	M1	M2	M3	M4	M5	Vamorolone
Dose Level Group 3	34.42	1.16	1.21	37.84	2.73	22.64

"Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug.~Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group" (NCT02760264)
Timeframe: Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.

Pharmacokinetic (PK) Assessments (AUC Inf)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. AUC inf= Area under the concentration vs. time curve to time infinity. (NCT02760264)
Timeframe: Day 1, Week 2

Pharmacokinetic (PK) Assessments (Cmax)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay (NCT02760264)
Timeframe: Day 1, Week 2

Pharmacokinetic (PK) Assessments (Tmax)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. tmax= time when plasma concentration is at maximum. (NCT02760264)
Timeframe: Day 1, Week 2

Pharmacokinetic (PK) Assessments CL (ml/hr/kg)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay (NCT02760264)
Timeframe: Day 1, Week 2

Pharmacokinetic (PK) Assessments t(1/2)

Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. t1/2= elimination half life. (NCT02760264)
Timeframe: Day 1, Week 2

Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide

Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose

Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin

Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin

Change From Baseline in 6MWD at Week 48

The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Participants with confirmed loss of ambulation at a particular visit were assigned a 6MWD result of 0. Baseline and Week 48 6MWD values are each the average of two valid 6MWD values, or a single available value if one was missing. (NCT01826487)
Timeframe: Baseline, Week 48

Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 48

"Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one's head, stand on one's heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal- achieves goal without any assistance. The sum of these scores (except for 'raise one's head' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0." (NCT01826487)
Timeframe: Baseline, Week 48

Change From Baseline in Time to Climb 4 Stairs at Week 48

"During the test for stair-climbing, the method of climbing used by the participant was categorized as follows: 1. Unable to up climb 4 standard stairs; 2. Climbs 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Climbs 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Climbs 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Climbs 4 standard stairs alternating feet, needs handrail for support; 6. Climbs 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported." (NCT01826487)
Timeframe: Baseline, Week 48

Change From Baseline in Time to Descend 4 Stairs at Week 48

"During the test for stair-descending, the method of descending used by the participant was categorized as follows: 1. Unable to descend 4 standard stairs; 2. Descends 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), using both arms on one or both handrails; 3. Descends 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), using one arm on one handrail; 4. Descends 4 standard stairs marking time (climbs one foot at a time, with both feet on a step before moving to next step), not needing handrail; 5. Descends 4 standard stairs alternating feet, needs handrail for support; 6. Descends 4 standard stairs alternating feet, not needing handrail support. A cumulative change from baseline data has been reported." (NCT01826487)
Timeframe: Baseline, Week 48

Change From Baseline in Time to Walk/Run 10 Meters at Week 48

During the test for walking/running 10 meters, the method of walk/run used by the participant was categorized as follows: 1. Unable to walk independently; 2. Unable to walk independently but can walk with support from a person or with assistive device (full leg calipers [knee-ankle-foot orthoses ] or walker); 3. Highly adapted gait, wide-based lordotic gait, cannot increase walking speed; 4. Moderately adapted gait, can pick up speed but cannot run; 5. Able to pick up speed but runs with a double stance phase (that is, cannot achieve both feet off the ground); 6. Runs and gets both feet off the ground (with no double stance phase). If the time taken to perform a test exceeded 30 seconds or if a participant could not perform the test due to disease progression, a value of 30 seconds was used. A cumulative change from baseline data has been reported. (NCT01826487)
Timeframe: Baseline, Week 48

Percentage of Participants With Treatment-Emergent Adverse Events (AEs)

An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. Treatment-emergent adverse event (TEAE) was defined as an adverse event that occurred or worsened in the period extending from first dose of study drug to 6 weeks after the last dose of study drug. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. (NCT01826487)
Timeframe: Baseline up to Week 54

Study Drug Compliance

Study drug compliance was assessed by quantification of used and unused study drug. Compliance was assessed in terms of the percentage of drug actually taken relative to the amount that should have been taken during the study. (NCT01826487)
Timeframe: Baseline to Week 48

Ataluren Plasma Concentration

Plasma samples for the determination of ataluren concentrations were analyzed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method with a lower limit of quantitation of 0.5 micrograms/milliliter (mcg/mL). (NCT01826487)
Timeframe: Weeks 8, 16, 24, 32, 40, and 48

Change From Baseline in PODCI Transfers/Basic Mobility and Sports/Physical Functioning Scores at Week 48

Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domains were prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Sports/Physical Functioning domain assesses difficulty encountered in participating in more active recreational activities. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain. (NCT01826487)
Timeframe: Baseline, Week 48

Time to 10 Percent (%) Persistent Worsening in 6MWD

The 6MWD test is a non-encouraged test performed in a 30 meters long flat corridor, where the participant is instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test. Time to 10% persistent worsening in 6MWD was defined as the last time that 6MWD was not 10% worse compared with baseline. Time to 10% persistent worsening in 6MWD <300 meters, >=300 to 400 meters, and >=400 meters was evaluated. For participants who did not have 10% 6MWD worsening or who were removed from study, time to 10% 6MWD worsening was censored at the time of the last 6MWD test. Participants who became non-ambulatory were considered to have 10% worsening. (NCT01826487)
Timeframe: Baseline to Week 48

Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 48, as Assessed by a Standardized Survey Administered by Site Personnel

Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 48), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much worse), 2 (slightly worse), 3 (unchanged), 4 (slightly better), or 5 (much better). (NCT01826487)
Timeframe: Baseline, Week 48

Muscle Imaging

MRI of leg muscles to measure changes in muscle fat percentage. The data point was collected by taking fat percentage at 6 months minus fat percentage at baseline with the following equation: (([final fat percentage - initial fat percentage]/initial fat percentage) * 100%)). All participants were included, both ambulatory and nonambulatory, with all genetic subtypes included. Five participants didn't have an MRI scan at 6 months and therefore were not included. Muscles imaged were analyzed for muscle fat changes from baseline to 6 months. Data is limited in interpretation due to various muscle groups in both ambulatory and non-ambulatory patients. (NCT04054375)
Timeframe: Baseline, 6 months

10 Meter Run Timed

time in seconds to walk/run 10 meters , less time to run indicates greater motor function (NCT04054375)
Timeframe: Baseline, Month 6

6 Minute Walk Test

number of meters walked in 6 minute period. Higher values indicate more motor function. (NCT04054375)
Timeframe: Baseline, Month 6

Bone Density

"whole dexa body scan to assess bone density with Z scores (more negative z score indicates increased risk for fractures).~Z-score of 0 represents the population mean, and is the average bone density. Positive scores indicate greater bone density and negative scores indicate decreased bone density, which could be clinically correlated with osteoporosis." (NCT04054375)
Timeframe: Baseline, 6 months

Brooke Scale Score

upper extremity assessment, scoring between 1- 6, lower score indicates more upper extremity function (NCT04054375)
Timeframe: Baseline, Month 6

Creatine Kinase

units/L, 0-unlimited, higher scores indicate worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Fasting Glucose

mg/dL, 0-unlimited, higher score indicates worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Fasting Lipid Profile

cholesterol levels - mg/dL, higher levels indicate worse outcomes (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Functional Assessments - NSAD Change

"Northstar Assessment for Dysferlinopathy~- score out of 58, range from 0 to 58, higher score indicates greater functional ability." (NCT04054375)
Timeframe: Baseline, Month 6

Functional Assessments - Upper Limb Strength

"Grip strength of the total force (Newtons) in both hands.~Participants attempted 3 trials in the right hand that was then averaged to create a right-hand average force score.~Then, the participants attempted 3 trials in the left hand that was then averaged to create a left-hand average force score.~The right-hand average force score was added to the left-hand average force score to create a total grip strength score." (NCT04054375)
Timeframe: Baseline and 6 months

HbgA1c

% , 0-100, higher score indicates worse outcome (NCT04054375)
Timeframe: Baseline and 6 months (Final Visit)

Lean Mass %

whole body dexa scans to assess lean mass % (0- 100 %). Increase lean mass % is the desired outcome. (NCT04054375)
Timeframe: Baseline, 6 months

Muscle Strength Test

Manual motor testing of the right knee flexion muscle group. (NCT04054375)
Timeframe: baseline, 6 months

Respiratory Changes

Force Vital Capacity (% of predicted value), decrease in FVC indicates declining respiratory function. (NCT04054375)
Timeframe: Baseline, 6 months

Vignos Scale Score

Lower extremity assessment, score from 1-10, lower score indicates more function. (NCT04054375)
Timeframe: Baseline, Month 6

Bayley III Gross Motor Scaled Score (Change From Baseline to 12 Month)

Bayley III Gross Motor Scaled Score measures motor development. This is normed for typically developing children and follow a bell shaped curve. The scale has mean of 10 +/-3 for children at all ages and is bell shaped. Therefore the two standard deviation range is 16 to 4 with higher values indicated better performance. Lower values have been shown to be common in boys with DMD and it this study the baseline average score was 4.2. (NCT02167217)
Timeframe: One year

Expression of GALGT2 as Demonstrated by Immunofluorescent Staining With Anti-CT Epitope Antibodies or WFA Lectin in Muscle Biopsy Sections at 120 Days Post Injection (Cohort 1) and 90 Days Post-injection (Cohort 2).

Percentage of fibers expressing GALGT2 in each biopsy sample. (NCT03333590)
Timeframe: Day 90 (Cohort 2) and Day 120 (Cohort 1)

GALGT2 Protein Expression Quantified by Western Blot and Assessed by Densitometry in Muscle Biopsy Tissue at 120 Days Post-injection (Cohort 1) and 90 Days Post-injection (Cohort 2)

Number of Unanticipated Grade III or Higher Treatment-Related Toxicities

Number of Meters Walked During the 6 Minute Walk Test

(NCT03333590)
Timeframe: Day 90 (Cohort 2) and Day 120 (Cohort 1) and Day 180 for both cohorts

Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).

The NSAA provides a score between 0 and 34 where higher numbers represent greater muscle function. (NCT03333590)
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1) and both Cohorts at Day 180, Months 12, 18 and 24

Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.

(NCT03333590)
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1) and both Cohorts at Day 180, Months 12, 18 and 24

Time Taken to Walk 100 Meters During the 100 Meter Walk Test.

(NCT03333590)
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1); both Cohorts at Day 180, Months 12, 18 and Cohort 2 at Month 24

Time Taken to Walk 100 Meters During the 100 Meter Walk Test.

(NCT03333590)
Timeframe: Days 90 (Cohort 2), 120 (Cohort 1); both Cohorts at Day 180, Months 12, 18 and Cohort 2 at Month 24

Efficacy: Change in Time to Complete a 100 Meter Timed Test.

The determination of whether spironolactone has similar efficacy to glucocorticoids in improving muscle strength in steroid naïve DMD patients. This will be determined by measuring the time to complete a 100 meter timed test (100M). (NCT03777319)
Timeframe: 6 months

Efficacy: Dynamometry Score

Secondary outcome measures will be Dynamometry score, which is a summation of maximum voluntary isometric contraction test values for knee flexion, knee extension, elbow flexion, and elbow extension (NCT03777319)
Timeframe: 6 months

Safety Will be Monitored Through Regular Review of Electrolytes.

Electrolytes (Sodium, Potassium, Cloride and Carbon dioxide, mmol/L) will be measured on a monthly basis following initiation of either spironolactone or prednisolone. (NCT03777319)
Timeframe: 6 months

Intervention	Number of Events (Number)
	Total Number of Adverse Events	Total Number of Treatment Emergent Adverse Events	Any Treatment Emergent Adverse Events	Any Drug Related Treatment Emergent Adverse Events	Any CTCAE Grade 3 or Higher TEAE	Discontinuation of Study Drug due to TEAE	Any Serious Treatment Emergent Adverse Events	Death
Dose Level Group 1	16	13	7	1	0	0	0	0
Dose Level Group 2	18	13	6	2	0	0	0	0
Dose Level Group 3	13	11	8	2	0	0	0	0
Dose Level Group 4	11	9	7	3	0	0	0	0

Intervention	[(hr)(ng)/mL] (Mean)
	Day 1	Week 2
Dose Level Group 1	118	164
Dose Level Group 2	379	544
Dose Level Group 3	761	1138
Dose Level Group 4	3279	3606

Intervention	ng/mL (Mean)
	Baseline	Day 1	Day 1 Change from Baseline	Week 2	Week 2 Change from Baseline	Week 4	Week 4 Change from Baseline
Dose Level Group 1	555.8	474.0	-81.8	443.8	-112.0	573.8	18.1
Dose Level Group 2	480.7	443.7	-34.5	407.8	-70.6	496.7	21.3
Dose Level Group 3	508.2	417.1	-91.1	346.6	-161.6	492.0	-16.2
Dose Level Group 4	511.5	475.2	-36.5	303.7	-207.8	566.3	54.8

Intervention	% change from Baseline (Mean)
	Day 1 Percent Change from Baseline	Week 2 Percent Change from Baseline	Week 4 Percent Change from Baseline
Dose Level Group 1	-12.2	-17.5	2.8
Dose Level Group 2	-5.4	-11.2	7.8
Dose Level Group 3	-17.4	-30.9	-1.4
Dose Level Group 4	-5.7	-39.9	11.8

Intervention	mcg/mL (Mean)
	Week 08	Week 16	Week 24	Week 32	Week 40	Week 48
Ataluren	4.230	3.429	3.323	3.480	3.997	3.544

Intervention	units on a scale (Mean)
	Transfers/Basic Mobility Score	Sports/Physical Functioning Score
Ataluren	-6.6	-5.6
Placebo	-8.8	-7.3

Intervention	days (Median)
	<300 meters	>=300 to <400 meters	>=400 meters
Ataluren	164	NA	NA
Placebo	56	280	NA

Intervention	Participants (Count of Participants)
	Physical Functioning: Upper Extremity Activity71971848					Physical Functioning: Upper Extremity Activity71971847	Physical Functioning: Walking71971848	Physical Functioning: Walking71971847	Physical Functioning: Climbing Stairs71971847	Physical Functioning: Climbing Stairs71971848	Physical Functioning: Other71971847	Physical Functioning: Other71971848	Cognition/Social Functioning71971848	Cognition/Social Functioning71971847	Emotional/Social Functioning71971847	Emotional/Social Functioning71971848	General Energy Level71971848	General Energy Level71971847	Sleep71971847	Sleep71971848	Other71971848	Other71971847
	Much better	Slightly better	Unchanged	Slightly worse	Much worse
Ataluren	7
Placebo	67
Ataluren	73
Placebo	6
Placebo	1
Placebo	5
Placebo	13
Ataluren	16
Placebo	57
Ataluren	60
Placebo	19
Ataluren	21
Placebo	18
Ataluren	4
Placebo	8
Placebo	61
Ataluren	65
Placebo	17
Ataluren	15
Placebo	9
Ataluren	10
Placebo	38
Ataluren	44
Placebo	7
Ataluren	5
Placebo	12
Ataluren	20
Placebo	71
Ataluren	74
Ataluren	3
Ataluren	2
Placebo	75
Ataluren	68
Placebo	2
Ataluren	8
Ataluren	12
Placebo	54
Ataluren	63
Placebo	11
Placebo	3
Placebo	4
Placebo	73
Ataluren	76
Placebo	0
Ataluren	1
Ataluren	6
Placebo	15
Ataluren	13
Ataluren	0

Intervention	meters (Number)
	Day 90 (Cohort 2) /Day 120 (Cohort 1)	Day 180
Cohort 1 (Minimal Efficacious Dose)	320	324
Cohort 2 (Minimal Efficacious Dose)	405	416

Intervention	score on a scale (Number)
	Day 90/Day 120	Day 180	Month 12	Month 18	Month 24
Cohort 1 (Minimal Efficacious Dose)	16	14	10	6	2
Cohort 2	21	23	23	23	23

Intervention	kg (Number)
	Day 90/Day 120-Right Knee Extension	Day 90/Day 120-Right Knee Flexion	Day 90/Day 120- Left Knee Extension	Day 90/Day 120-Left Knee Flexion	Day 180-Right Knee Extension	Day 180-Right Knee Flexion	Day 180-Left Knee Extension	Day 180-Left Knee Flexion	Month 12-Right Knee Extension	Month 12-Right Knee Flexion	Month 12-Left Knee Extension	Month 12-Left Knee Flexion	Month 18-Right Knee Extension	Month 18-Right Knee Flexion	Month 18-Left Knee Extension	Month 18-Left Knee Flexion	Month 24-Right Knee Extension	Month 24-Right Knee Flexion	Month 24-Left Knee Extension	Month 24-Left Knee Flexion
Cohort 1 (Minimal Efficacious Dose)	7.42	6.06	8.78	6.12	7.13	6.1	8.66	6.69	7.49	5.67	7.5	5.32	4.55	6.11	4.96	6.26	5.06	4.41	6.93	4.17
Cohort 2	7.04	8.12	5.9	8.4	9.73	4.24	8.19	5.25	9.85	5.85	8.02	5.12	7.67	6.89	7.34	6.08	9.81	5.04	5.21	4.87

Intervention	kg (Number)
	Elbow Flexion (Right)-Baseline	Elbow Flexion (Left)-Baseline	Elbow Extension (Right)-Baseline	Elbow Extension (Left)-Baseline	Knee Flexion (Right)-Baseline	Knee Flexion (Left)-Baseline	Knee Extension (Right)-Baseline	Knee Extension (Left)-Baseline	Elbow Flexion (Right)-Month 6	Elbow Flexion (Left)-Month 6	Elbow Extension (Right)-Month 6	Elbow Extension (Left)-Month 6	Knee Flexion (Right)-Month 6	Knee Flexion (Left)-Month 6	Knee Extension (Right)-Month 6	Knee Extension (Left)-Month 6
Prednisolone	3.6	4.1	5.3	4.1	3.3	3.4	4.8	5.2	2.9	3.4	4.3	3.8	4.1	3.9	6	5.1
Spironolactone	0	0	0	0	4.1	2.8	3.8	5.9	3.1	3.5	2.4	2.5	4.3	4.1	7.2	8.3

Intervention	mmol/L (Number)
	Sodium-Baseline	Sodium-Month 1	Sodium-Month 2	Sodium-Month 3	Sodium-Month 4	Sodium-Month 5	Sodium-Month 6	Potassium-Baseline	Potassium-Month 1	Potassium-Month 2	Potassium-Month 3	Potassium-Month 4	Potassium-Month 5	Potassium-Month 6	Chloride-Baseline	Chloride-Month 1	Chloride-Month 2	Chloride-Month 3	Chloride-Month 4	Chloride-Month 5	Chloride-Month 6	CO2-Baseline	CO2-Month 1	CO2-Month 2	CO2-Month 3	CO2-Month 4	CO2-Month 5	CO2-Month 6
Prednisolone	140	140	139	141	139	139	143	3.8	4	4.5	3.9	4.6	4.2	3.9	105	105	104	105	105	106	105	22	24	24	24	25	26	26
Spironolactone	142	142	141	142	139	139	140	4.5	4.7	4.2	4.1	4.5	4.5	4.3	103	109	107	103	103	103	101	29	22	25	27	28	28	26

Article	Year
Biomaterial investigations in dentistry, 2021, Volume: 8, Issue:1 Topics: Adult; Alcohol Drinking; Ammonia; Animals; Anti-Inflammatory Agents; Area Under Curve; B-Lymphocytes	2021
Prednisone and deflazacort in Duchenne muscular dystrophy: a patient perspective and plain language summary publication of the Cincinnati study. Journal of comparative effectiveness research, 2022, Volume: 11, Issue:11 Topics: Child; Humans; Inflammation; Language; Male; Muscular Dystrophy, Duchenne; Prednisone; Pregnenedione	2022
Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy. Journal of neuromuscular diseases, 2022, Volume: 9, Issue:4 Topics: Humans; Muscular Dystrophy, Duchenne; Prednisolone; Prednisone; Pregnenediones; Prospective Studies	2022
Microbes, metabolites and muscle: Is the gut-muscle axis a plausible therapeutic target in Duchenne muscular dystrophy? Experimental physiology, 2023, Volume: 108, Issue:9 Topics: Animals; Glucocorticoids; Inflammation; Mice; Mice, Inbred mdx; Muscle, Skeletal; Muscular Dystrophy	2023
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials. Muscle & nerve, 2020, Volume: 61, Issue:1 Topics: Anti-Inflammatory Agents; Child; Disease Progression; Humans; Male; Multicenter Studies as Topic; Mu	2020
The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy. Journal of managed care & specialty pharmacy, 2020, Volume: 26, Issue:4 Topics: Cost-Benefit Analysis; Dystrophin; Exons; Humans; Immunosuppressive Agents; Models, Economic; Morpho	2020
Pulmonary function and clinical correlation in DMD. Paediatric respiratory reviews, 2019, Volume: 30 Topics: Disease Progression; Glucocorticoids; Humans; Morpholinos; Muscular Dystrophy, Duchenne; Oxadiazoles	2019
Update in Duchenne and Becker muscular dystrophy. Current opinion in neurology, 2019, Volume: 32, Issue:5 Topics: Adrenal Cortex Hormones; Dystrophin; Exons; Genetic Therapy; Humans; Male; Muscular Dystrophy, Duche	2019
Corticosteroids for the treatment of Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2016, May-05, Issue:5 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscle Strength; Muscular Dystrophy, Duchenn	2016
Interventions for increasing ankle range of motion in patients with neuromuscular disease. The Cochrane database of systematic reviews, 2010, Feb-17, Issue:2 Topics: Ankle Joint; Charcot-Marie-Tooth Disease; Equinus Deformity; Glucocorticoids; Humans; Male; Muscular	2010
Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. Journal of child neurology, 2010, Volume: 25, Issue:9 Topics: Child; Disease Progression; Drug Administration Schedule; Humans; Male; Muscular Dystrophy, Duchenne	2010
Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives. Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2012, Volume: 31, Issue:1 Topics: Aminoglycosides; Genetic Therapy; Glucocorticoids; Humans; Immunosuppressive Agents; Muscular Dystro	2012
Old and new therapeutic developments in steroid treatment in Duchenne muscular dystrophy. Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2012, Volume: 31, Issue:1 Topics: Exercise Test; Glucocorticoids; Humans; Immunosuppressive Agents; Muscle Strength; Muscular Dystroph	2012
Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy. Physical medicine and rehabilitation clinics of North America, 2012, Volume: 23, Issue:4 Topics: Drug Discovery; Glucocorticoids; Humans; Muscular Dystrophy, Duchenne; Prednisone; Pregnenediones	2012
Deflazacort for the treatment of Duchenne Dystrophy: a systematic review. BMC neurology, 2003, Sep-08, Volume: 3 Topics: Adolescent; Anti-Inflammatory Agents; Child; Child, Preschool; Humans; Immunosuppressive Agents; Mal	2003
Glucocorticoid corticosteroids for Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2004, Issue:2 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Prednisolone;	2004
Glucocorticoid corticosteroids for Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2008, Jan-23, Issue:1 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Prednisolone;	2008
Glucocorticoid corticosteroids for Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2008, Jan-23, Issue:1 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Prednisolone;	2008
Glucocorticoid corticosteroids for Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2008, Jan-23, Issue:1 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Prednisolone;	2008
Glucocorticoid corticosteroids for Duchenne muscular dystrophy. The Cochrane database of systematic reviews, 2008, Jan-23, Issue:1 Topics: Adrenal Cortex Hormones; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Prednisolone;	2008
Muscular dystrophy. Pediatrics in review, 2000, Volume: 21, Issue:7 Topics: Child; Glucocorticoids; Humans; Male; Muscles; Muscular Dystrophy, Duchenne; Prednisone	2000
Pharmacologic and genetic therapy for childhood muscular dystrophies. Current neurology and neuroscience reports, 2001, Volume: 1, Issue:2 Topics: Animals; Cell Transplantation; Child; Child, Preschool; Clinical Trials as Topic; Creatine; Cytoskel	2001
Corticosteroids in Duchenne muscular dystrophy: a reappraisal. Journal of child neurology, 2002, Volume: 17, Issue:3 Topics: Adolescent; Anti-Inflammatory Agents; Child; Child, Preschool; Cushing Syndrome; Evidence-Based Medi	2002

Article	Year
Direct costs of adhering to selected Duchenne muscular dystrophy Care Considerations: Estimates from a midwestern state. Muscle & nerve, 2022, Volume: 65, Issue:5 Topics: Adolescent; Adult; Child; Child, Preschool; Humans; Muscular Dystrophy, Duchenne; Prednisone; Young	2022
No difference in postoperative complication rates or cardiopulmonary function for early versus late scoliosis correction in Duchenne muscular dystrophy. Spine deformity, 2022, Volume: 10, Issue:6 Topics: Child; Humans; Muscular Dystrophy, Duchenne; Postoperative Complications; Prednisone; Retrospective	2022
Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy1. Journal of neuromuscular diseases, 2023, Volume: 10, Issue:1 Topics: Adolescent; Disease Progression; Humans; Longitudinal Studies; Male; Muscular Dystrophy, Duchenne; P	2023
A Mixed-Method Study Exploring Patient-Experienced and Caregiver-Reported Benefits and Side Effects of Corticosteroid Use in Duchenne Muscular Dystrophy. Journal of neuromuscular diseases, 2023, Volume: 10, Issue:4 Topics: Adrenal Cortex Hormones; Adult; Caregivers; Glucocorticoids; Humans; Muscular Dystrophy, Duchenne; P	2023
Dimethyl fumarate modulates the dystrophic disease program following short-term treatment. JCI insight, 2023, Nov-08, Volume: 8, Issue:21 Topics: Animals; Dimethyl Fumarate; Mice; Mice, Inbred mdx; Muscles; Muscular Dystrophy, Duchenne; Prednison	2023
Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy. Journal of neuromuscular diseases, 2023, Volume: 10, Issue:6 Topics: Anti-Inflammatory Agents; Glucocorticoids; Humans; Muscular Dystrophy, Duchenne; Prednisone; Pregnad	2023
Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming. JCI insight, 2019, 12-19, Volume: 4, Issue:24 Topics: Anacardic Acids; Animals; Biomarkers; Child; Cross-Sectional Studies; Disease Models, Animal; Drug T	2019
Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center. Journal of comparative effectiveness research, 2020, Volume: 9, Issue:3 Topics: Adolescent; Bone Density; Child; Child, Preschool; Humans; Male; Muscular Dystrophy, Duchenne; Predn	2020
Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania. Brain & development, 2020, Volume: 42, Issue:3 Topics: Adolescent; Adult; Child; Child, Preschool; China; Health Care Surveys; Humans; Infant; Japan; Male;	2020
Can photobiomodulation therapy be an alternative to pharmacological therapies in decreasing the progression of skeletal muscle impairments of mdx mice? PloS one, 2020, Volume: 15, Issue:8 Topics: Animals; Anti-Inflammatory Agents, Non-Steroidal; Combined Modality Therapy; Disease Models, Animal;	2020
Quadriceps muscle strength in Duchenne muscular dystrophy and effect of corticosteroid treatment. Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology, 2020, Volume: 39, Issue:4 Topics: Adolescent; Age Factors; Anti-Inflammatory Agents; Child; Child, Preschool; Drug Administration Sche	2020
Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment. Muscle & nerve, 2021, Volume: 64, Issue:5 Topics: Clinical Trials, Phase III as Topic; Humans; Muscular Dystrophy, Duchenne; Prednisolone; Prednisone;	2021
Intermittent glucocorticoid steroid dosing enhances muscle repair without eliciting muscle atrophy. The Journal of clinical investigation, 2017, Jun-01, Volume: 127, Issue:6 Topics: Animals; Annexin A6; Cells, Cultured; Drug Administration Schedule; Drug Evaluation, Preclinical; Ge	2017
Intermittent Glucocorticoid Dosing Improves Muscle Repair and Function in Mice with Limb-Girdle Muscular Dystrophy. The American journal of pathology, 2017, Volume: 187, Issue:11 Topics: Animals; Dystrophin; Glucocorticoids; Membrane Proteins; Mice; Muscle, Skeletal; Muscular Dystrophie	2017
How glucocorticoids change life in Duchenne muscular dystrophy. Lancet (London, England), 2018, 02-03, Volume: 391, Issue:10119 Topics: Glucocorticoids; Humans; Muscular Dystrophy, Duchenne; Prednisone	2018
Effect of 25-HydroxyVitamin D Deficiency and Its Interaction with Prednisone Treatment on Musculoskeletal Health in Growing Mdx Mice. Calcified tissue international, 2018, Volume: 103, Issue:3 Topics: Animals; Anti-Inflammatory Agents; Bone and Bones; Bone Density; Male; Mice; Mice, Inbred mdx; Muscl	2018
Cataract development associated with long-term glucocorticoid therapy in Duchenne muscular dystrophy patients. Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus, 2018, Volume: 22, Issue:3 Topics: Adolescent; Cataract; Cataract Extraction; Child; Female; Glucocorticoids; Humans; Immunosuppressive	2018
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. Physiological genomics, 2018, 09-01, Volume: 50, Issue:9 Topics: Animals; Base Sequence; Chronic Disease; Disease Models, Animal; Gene Expression Regulation; Inflamm	2018
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. Physiological genomics, 2018, 09-01, Volume: 50, Issue:9 Topics: Animals; Base Sequence; Chronic Disease; Disease Models, Animal; Gene Expression Regulation; Inflamm	2018
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. Physiological genomics, 2018, 09-01, Volume: 50, Issue:9 Topics: Animals; Base Sequence; Chronic Disease; Disease Models, Animal; Gene Expression Regulation; Inflamm	2018
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone. Physiological genomics, 2018, 09-01, Volume: 50, Issue:9 Topics: Animals; Base Sequence; Chronic Disease; Disease Models, Animal; Gene Expression Regulation; Inflamm	2018
Myocarditis in Duchenne Muscular Dystrophy After Changing Steroids. JAMA cardiology, 2018, 10-01, Volume: 3, Issue:10 Topics: Adolescent; Drug Substitution; Emergency Service, Hospital; Humans; Male; Muscular Dystrophy, Duchen	2018
Comparison of Pulmonary Function Decline in Steroid-Treated and Steroid-Naïve Patients with Duchenne Muscular Dystrophy. The Journal of pediatrics, 2019, Volume: 210 Topics: Adolescent; Child; Disease Progression; Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne;	2019
Levels of α7 integrin and laminin-α2 are increased following prednisone treatment in the mdx mouse and GRMD dog models of Duchenne muscular dystrophy. Disease models & mechanisms, 2013, Volume: 6, Issue:5 Topics: Animals; Antigens, CD; Disease Models, Animal; Dogs; Gene Expression Regulation; Humans; Integrin al	2013
Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect. Human gene therapy, 2013, Volume: 24, Issue:9 Topics: Age Factors; CD4-Positive T-Lymphocytes; CD8-Positive T-Lymphocytes; Dependovirus; Dystrophin; Genet	2013
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy. The Journal of cell biology, 2014, Oct-13, Volume: 207, Issue:1 Topics: Animals; Anti-Inflammatory Agents; Cell Differentiation; Cells, Cultured; Dystrophin; Fibrosis; Huma	2014
Corticosteroid Treatments in Males With Duchenne Muscular Dystrophy: Treatment Duration and Time to Loss of Ambulation. Journal of child neurology, 2015, Volume: 30, Issue:10 Topics: Adrenal Cortex Hormones; Age of Onset; Child; Disease Progression; Follow-Up Studies; Humans; Longit	2015
Myocardial fibrosis burden predicts left ventricular ejection fraction and is associated with age and steroid treatment duration in duchenne muscular dystrophy. Journal of the American Heart Association, 2015, Mar-26, Volume: 4, Issue:4 Topics: Adolescent; Adult; Age Factors; Child; Disease Progression; Fibrosis; Glucocorticoids; Heart; Humans	2015
Chronic Dosing with Membrane Sealant Poloxamer 188 NF Improves Respiratory Dysfunction in Dystrophic Mdx and Mdx/Utrophin-/- Mice. PloS one, 2015, Volume: 10, Issue:8 Topics: Animals; Anti-Inflammatory Agents; Diaphragm; Disease Models, Animal; Dose-Response Relationship, Dr	2015
Corticosteroid Treatment and Growth Patterns in Ambulatory Males with Duchenne Muscular Dystrophy. The Journal of pediatrics, 2016, Volume: 173 Topics: Adolescent; Body Height; Body Mass Index; Body Weight; Case-Control Studies; Child; Child, Preschool	2016
Identifying evidence of cardio-renal syndrome in patients with Duchenne muscular dystrophy using cystatin C. Neuromuscular disorders : NMD, 2016, Volume: 26, Issue:10 Topics: Adolescent; Adult; Anti-Inflammatory Agents; Biomarkers; Cardio-Renal Syndrome; Child; Cross-Section	2016
Long-Term Outcome of Interdisciplinary Management of Patients with Duchenne Muscular Dystrophy Receiving Daily Glucocorticoid Treatment. The Journal of pediatrics, 2017, Volume: 182 Topics: Adolescent; Age Factors; Child; Cohort Studies; Dose-Response Relationship, Drug; Drug Administratio	2017
Prednisone 10 days on/10 days off in patients with Duchenne muscular dystrophy. Journal of neurology, 2009, Volume: 256, Issue:5 Topics: Adolescent; Age Factors; Age of Onset; Akathisia, Drug-Induced; Anti-Inflammatory Agents; Child; Chi	2009
Corticosteroid effects on blood gene expression in Duchenne muscular dystrophy. The pharmacogenomics journal, 2009, Volume: 9, Issue:6 Topics: Adipose Tissue; Adolescent; Case-Control Studies; Child; Child, Preschool; Chondroitin Sulfates; Gen	2009
Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular Dystrophy. PloS one, 2010, Jun-21, Volume: 5, Issue:6 Topics: Animals; Arginine; Behavior, Animal; Butyrates; Disease Models, Animal; Drug Therapy, Combination; F	2010
Duchenne muscular dystrophy: the effect of glucocorticoids on ventilator use and ambulation. American journal of physical medicine & rehabilitation, 2010, Volume: 89, Issue:8 Topics: Adult; Age Factors; Child; Glucocorticoids; Humans; Mobility Limitation; Muscular Dystrophy, Duchenn	2010
Non-invasive optical imaging of muscle pathology in mdx mice using cathepsin caged near-infrared imaging. Molecular imaging and biology, 2011, Volume: 13, Issue:3 Topics: Animals; Cathepsin B; Diagnostic Imaging; Hindlimb; Humans; Infrared Rays; Injections, Intraperitone	2011
Impact of bisphosphonates on survival for patients with Duchenne muscular dystrophy. Pediatrics, 2011, Volume: 127, Issue:2 Topics: Adolescent; Adult; Child; Cohort Studies; Diphosphonates; Drug Therapy, Combination; Follow-Up Studi	2011
Nitric oxide donors improve prednisone effects on muscular dystrophy in the mdx mouse diaphragm. American journal of physiology. Cell physiology, 2011, Volume: 300, Issue:5 Topics: Animals; Body Weight; Calcinosis; Diaphragm; Female; Guaifenesin; Isosorbide Dinitrate; Male; Mice;	2011
Weekend high-dosage prednisone: a new option for treatment of Duchenne muscular dystrophy. Neurology, 2011, Aug-02, Volume: 77, Issue:5 Topics: Dose-Response Relationship, Drug; Drug Administration Schedule; Glucocorticoids; Humans; Muscular Dy	2011
Normal height and weight in a series of ambulant Duchenne muscular dystrophy patients using the 10 day on/10 day off prednisone regimen. Neuromuscular disorders : NMD, 2012, Volume: 22, Issue:6 Topics: Body Height; Body Weight; Child; Child, Preschool; Drug Administration Schedule; Follow-Up Studies;	2012
Early corticosteroid treatment in 4 Duchenne muscular dystrophy patients: 14-year follow-up. Muscle & nerve, 2012, Volume: 45, Issue:6 Topics: Adolescent; Adrenal Cortex Hormones; Follow-Up Studies; Humans; Longitudinal Studies; Male; Muscular	2012
Growth hormone treatment in boys with Duchenne muscular dystrophy and glucocorticoid-induced growth failure. Neuromuscular disorders : NMD, 2012, Volume: 22, Issue:12 Topics: Body Height; Body Weight; Child; Glucocorticoids; Growth Disorders; Human Growth Hormone; Humans; Ma	2012
Health-related quality of life in children and adolescents with Duchenne muscular dystrophy. Pediatrics, 2012, Volume: 130, Issue:6 Topics: Activities of Daily Living; Adolescent; Age Factors; Anti-Inflammatory Agents; Child; Child, Prescho	2012
24 month longitudinal data in ambulant boys with Duchenne muscular dystrophy. PloS one, 2013, Volume: 8, Issue:1 Topics: Adolescent; Analysis of Variance; Anti-Inflammatory Agents; Child; Child, Preschool; Exercise Test;	2013
Bone mineral density and bone metabolism in Duchenne muscular dystrophy. Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA, 2003, Volume: 14, Issue:9 Topics: 25-Hydroxyvitamin D 2; Absorptiometry, Photon; Adolescent; Anthropometry; Bone and Bones; Bone Densi	2003
New treatment alternatives for Duchenne and Becker muscular dystrophy. Neurology, 2004, Mar-23, Volume: 62, Issue:6 Topics: Administration, Oral; Adrenergic beta-Agonists; Albuterol; Child; Child, Preschool; Clinical Trials	2004
Prednisone reduces muscle degeneration in dystrophin-deficient Caenorhabditis elegans. Neuromuscular disorders : NMD, 2004, Volume: 14, Issue:6 Topics: Animals; Anti-Inflammatory Agents; Caenorhabditis elegans; Caenorhabditis elegans Proteins; Disease	2004
Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology, 2005, Jan-11, Volume: 64, Issue:1 Topics: Adolescent; Child; Child, Preschool; Creatinine; Dose-Response Relationship, Drug; Drug Administrati	2005
Prednisone for Duchenne muscular dystrophy. The Lancet. Neurology, 2005, Volume: 4, Issue:5 Topics: Child, Preschool; Clinical Trials as Topic; Dose-Response Relationship, Drug; Drug Administration Sc	2005
Muscular dystrophies. Pediatric annals, 2005, Volume: 34, Issue:7 Topics: Adolescent; Cardiomyopathies; Child; Humans; Mobility Limitation; Muscular Dystrophy, Duchenne; Pedi	2005
Corticosteroid treatment and functional improvement in Duchenne muscular dystrophy: long-term effect. American journal of physical medicine & rehabilitation, 2005, Volume: 84, Issue:11 Topics: Adolescent; Anti-Inflammatory Agents; Body Height; Body Weight; Disease Progression; Dose-Response R	2005
[Guideline on the use of corticosteroids in Duchenne muscular dystrophy from paediatric neurologists, neurologists and rehabilitation physicians]. Nederlands tijdschrift voor geneeskunde, 2006, Mar-25, Volume: 150, Issue:12 Topics: Glucocorticoids; Humans; Male; Muscular Dystrophy, Duchenne; Netherlands; Practice Guidelines as Top	2006
The glucocorticoid receptor N363S polymorphism and steroid response in Duchenne dystrophy. Journal of neurology, neurosurgery, and psychiatry, 2006, Volume: 77, Issue:10 Topics: Anti-Inflammatory Agents; Child; Child, Preschool; Cohort Studies; Female; Humans; Male; Muscular Dy	2006
The effects of glucocorticoid therapy on the inflammatory and dendritic cells in muscular dystrophies. International journal of experimental pathology, 2006, Volume: 87, Issue:6 Topics: Adolescent; Adult; Analysis of Variance; Biopsy; Child; Child, Preschool; Dendritic Cells; Female; G	2006
Effect of long-term steroids on cough efficiency and respiratory muscle strength in patients with Duchenne muscular dystrophy. Pediatrics, 2007, Volume: 119, Issue:2 Topics: Adolescent; Case-Control Studies; Child; Cough; Glucocorticoids; Humans; Muscle Strength; Muscular D	2007
Quantification of muscle strength and motor ability in patients with Duchenne muscular dystrophy on steroid therapy. Arquivos de neuro-psiquiatria, 2007, Volume: 65, Issue:2A Topics: Anti-Inflammatory Agents; Child; Child, Preschool; Female; Follow-Up Studies; Glucocorticoids; Human	2007
Bone mineral density in children exposed to chronic glucocorticoid therapy. Clinical pediatrics, 2008, Volume: 47, Issue:5 Topics: Adolescent; Arthritis, Juvenile; Bone Density; Calcium, Dietary; Child; Child, Preschool; Cross-Sect	2008
Corticosteroid treatment retards development of ventricular dysfunction in Duchenne muscular dystrophy. Neuromuscular disorders : NMD, 2008, Volume: 18, Issue:5 Topics: Adolescent; Adrenal Cortex Hormones; Cardiomyopathies; Child; Echocardiography; Follow-Up Studies; G	2008
Prednisone therapy in Becker's muscular dystrophy. Journal of child neurology, 2001, Volume: 16, Issue:11 Topics: Adolescent; Anti-Inflammatory Agents; Child; Creatine Kinase; Dystrophin; Humans; Male; Muscle, Skel	2001