Page last updated: 2024-08-23

miglustat and Disease Exacerbation

miglustat has been researched along with Disease Exacerbation in 21 studies

Research

Studies (21)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's6 (28.57)29.6817
2010's14 (66.67)24.3611
2020's1 (4.76)2.80

Authors

AuthorsStudies
Ardissone, A; Caciotti, A; Calamai, M; Casini, A; Deodato, F; Di Rocco, M; Fischetto, R; Fiumara, A; Guerrini, R; Mancardi, MM; Mangone, G; Marangi, A; Morrone, A; Parini, R; Pavone, FS; Procopio, E; Ricci, S; Salviati, A; Strisciuglio, P; Tonin, R1
Barrio, SC; Extremera, VC; Gutiérrez-Solana, LG; Jiménez, IB; la Concepción Fournier Del Castillo, M; Marín, LL; Orduña, BE1
Stellitano, LA; Verity, CM; Winstone, AM1
Caciotti, A; Deodato, F; Dionisi-Vici, C; Donati, MA; Morrone, A; Procopio, E; Rampazzo, A; Scarpa, M; Taurisano, R1
Adachi, K; Itamura, S; Jimbo, E; Miyauchi, A; Nakamura, S; Nakano, Y; Nanba, E; Narita, A; Osaka, H; Usui, M; Yamagata, T1
Hawlitschka, A; Hovakimyan, M; Lukas, J; Maass, F; Petersen, J; Rolfs, A; Schmitt, O; Witt, M; Wree, A1
Cornelisse, P; Mengel, E; Muller, A; Patterson, MC; Pineda, M; Schwierin, B; Vanier, MT1
Abel, L; Bowman, EA; Desmond, P; Fahey, M; Velakoulis, D; Walterfang, M1
Audoin, B; Burzykowski, T; Chabrol, B; Galanaud, D; Kaphan, E; Sedel, F; Tourbah, A; Tranchant, C; Vanier, MT1
Ali, NF; Davidson, CD; Dobrenis, K; Micsenyi, MC; Ory, DS; Renault, S; Stephney, G; Vanier, MT; Walkley, SU1
Dekomien, G; Lefeber, DJ; Morava, E; Wevers, RA; Willemsen, MA; Wortmann, SB1
Berg, JE; Tallaksen, CM1
Guitton, J; Masciullo, M; Modoni, A; Ricci, E; Santoro, M; Silvestri, G; Tonali, P1
Fornfeist, S1
Guthoff, R; Hovakimyan, M; Lukas, J; Maass, F; Petersen, J; Reichard, M; Rolfs, A; Stachs, O; Witt, M; Wree, A1
Chien, YH; Huang, AC; Hwu, WL; Lee, NC; Peng, SF; Su, SC; Tsai, LK; Tseng, CC; Yang, CC1
Klünemann, HH; Santosh, PJ; Sedel, F1
Gordo, MM; Marfa, MP; Pérez-Poyato, MS1
Guerrera, M; Ladisch, S1
Alfonso, P; Capablo, JL; de Cabezón, AS; Franco, R; Giraldo, P; Pocovi, M1
Froissart, R; Guffon, N; Maire, I1

Reviews

2 review(s) available for miglustat and Disease Exacerbation

ArticleYear
Treatable metabolic psychoses that go undetected: what Niemann-Pick type C can teach us.
    International journal of psychiatry in clinical practice, 2012, Volume: 16, Issue:3

    Topics: 1-Deoxynojirimycin; Age of Onset; Ataxia; Diagnosis, Differential; Disease Progression; Enzyme Inhibitors; Genetic Testing; Humans; Magnetic Resonance Spectroscopy; Niemann-Pick Disease, Type C; Ophthalmoplegia; Prevalence; Psychotic Disorders; Splenomegaly; Treatment Outcome

2012
[Current development and usefulness of biomarkers for Gaucher disease follow up].
    La Revue de medecine interne, 2007, Volume: 28 Suppl 2

    Topics: 1-Deoxynojirimycin; Adult; Biomarkers; Chemokines, CC; Disease Progression; Enzyme Inhibitors; Female; Follow-Up Studies; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Hexosaminidases; Humans; Immunohistochemistry; Infant, Newborn; Peptidyl-Dipeptidase A; Pregnancy; Pregnancy Complications; Recombinant Proteins; Time Factors

2007

Other Studies

19 other study(ies) available for miglustat and Disease Exacerbation

ArticleYear
Pre-diagnosing and managing patients with GM1 gangliosidosis and related disorders by the evaluation of GM1 ganglioside content.
    Scientific reports, 2019, 11-27, Volume: 9, Issue:1

    Topics: 1-Deoxynojirimycin; Biomarkers; Cells, Cultured; Disease Progression; Female; Fibroblasts; Flow Cytometry; G(M1) Ganglioside; Gangliosidosis, GM1; Glycoside Hydrolase Inhibitors; Humans; Lymphocytes; Male; Optical Imaging; Phenotype; Severity of Illness Index

2019
Long-term normalization of cognitive and psychopathological alterations in a juvenile Niemann-Pick type C case.
    Neurodegenerative disease management, 2020, Volume: 10, Issue:2

    Topics: 1-Deoxynojirimycin; Adolescent; Child; Cognition; Disease Progression; Early Diagnosis; Female; Glycoside Hydrolase Inhibitors; Humans; Longitudinal Studies; Niemann-Pick Disease, Type C

2020
Niemann-Pick type C as a cause of progressive intellectual and neurological deterioration in childhood.
    Developmental medicine and child neurology, 2017, Volume: 59, Issue:9

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age of Onset; Brain; Child; Child, Preschool; Delayed Diagnosis; Disease Progression; Epidemiological Monitoring; Female; Follow-Up Studies; Glycoside Hydrolase Inhibitors; Humans; Infant; Intellectual Disability; Male; Niemann-Pick Disease, Type C; Prospective Studies; Time Factors; United Kingdom

2017
The treatment of juvenile/adult GM1-gangliosidosis with Miglustat may reverse disease progression.
    Metabolic brain disease, 2017, Volume: 32, Issue:5

    Topics: 1-Deoxynojirimycin; Adult; Age Factors; beta-Galactosidase; Cognition Disorders; Disease Progression; Female; Follow-Up Studies; Gait; Gangliosidosis, GM1; Genotype; Glycoside Hydrolase Inhibitors; Humans; Language Disorders; Motor Skills; Movement Disorders; Neuropsychological Tests; Treatment Outcome; Walking; Young Adult

2017
Miglustat therapy in a case of early-infantile Niemann-Pick type C.
    Brain & development, 2017, Volume: 39, Issue:10

    Topics: 1-Deoxynojirimycin; Base Sequence; Carrier Proteins; Child, Preschool; Developmental Disabilities; Disease Progression; Glucosyltransferases; Humans; Intracellular Signaling Peptides and Proteins; Japan; Male; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C

2017
Reduced cerebellar neurodegeneration after combined therapy with cyclodextrin/allopregnanolone and miglustat in NPC1: a mouse model of Niemann-Pick type C1 disease.
    Journal of neuroscience research, 2015, Volume: 93, Issue:3

    Topics: 1-Deoxynojirimycin; Animals; Cell Count; Cerebellum; Cyclodextrins; Disease Models, Animal; Disease Progression; Drug Therapy, Combination; Enzyme Inhibitors; Mice; Nerve Degeneration; Neurons; Niemann-Pick Disease, Type C; Pregnanolone

2015
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.
    Orphanet journal of rare diseases, 2015, May-28, Volume: 10

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Child, Preschool; Cohort Studies; Disease Progression; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Prospective Studies; Treatment Outcome; Young Adult

2015
Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat.
    Journal of neurology, 2015, Volume: 262, Issue:9

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; Caudate Nucleus; Cerebellum; Disease Progression; Enzyme Inhibitors; Female; Humans; Longitudinal Studies; Magnetic Resonance Imaging; Male; Middle Aged; Niemann-Pick Disease, Type C; Organ Size; Putamen; Thalamus; Young Adult

2015
Normalisation of brain spectroscopy findings in Niemann-Pick disease type C patients treated with miglustat.
    Journal of neurology, 2016, Volume: 263, Issue:5

    Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aspartic Acid; Brain; Choline; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Linear Models; Longitudinal Studies; Male; Niemann-Pick Disease, Type C; Patient Dropouts; Treatment Outcome; Young Adult

2016
Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression.
    PloS one, 2009, Sep-11, Volume: 4, Issue:9

    Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; beta-Cyclodextrins; Cholesterol; Cyclodextrins; Disease Models, Animal; Disease Progression; Drug Synergism; Enzyme Inhibitors; Glycosphingolipids; Mice; Mice, Transgenic; Neurons; Niemann-Pick Disease, Type C; Pregnanolone; Treatment Outcome

2009
Substrate deprivation therapy in juvenile Sandhoff disease.
    Journal of inherited metabolic disease, 2009, Volume: 32 Suppl 1

    Topics: 1-Deoxynojirimycin; Adolescent; Child, Preschool; Disease Progression; Enzyme Inhibitors; Glucosyltransferases; Hexosaminidase B; Humans; Male; Mutation; Sandhoff Disease

2009
Miglustat therapy in juvenile Sandhoff disease.
    Journal of inherited metabolic disease, 2009, Volume: 32 Suppl 1

    Topics: 1-Deoxynojirimycin; Adolescent; beta-Hexosaminidase beta Chain; Cerebellum; Child, Preschool; Depression; Disease Progression; Electroconvulsive Therapy; Enzyme Inhibitors; Glucosyltransferases; Humans; Male; Mutation; Sandhoff Disease

2009
Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up.
    Journal of inherited metabolic disease, 2010, Volume: 33 Suppl 3

    Topics: 1-Deoxynojirimycin; Disease Progression; Enzyme Inhibitors; Glucosyltransferases; Humans; Magnetic Resonance Imaging; Male; Middle Aged; Muscle Weakness; Muscular Atrophy, Spinal; Neurologic Examination; Predictive Value of Tests; Sandhoff Disease; Time Factors; Treatment Outcome

2010
[Niemann Pick type C disease].
    Kinderkrankenschwester : Organ der Sektion Kinderkrankenpflege, 2010, Volume: 29, Issue:9

    Topics: 1-Deoxynojirimycin; Brain; Child; Child, Preschool; Chromosome Aberrations; Combined Modality Therapy; Developmental Disabilities; Diagnosis, Differential; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Genes, Recessive; Genetic Testing; Humans; Infant, Newborn; Magnetic Resonance Imaging; Niemann-Pick Disease, Type C; Parenting

2010
Corneal alterations during combined therapy with cyclodextrin/allopregnanolone and miglustat in a knock-out mouse model of NPC1 disease.
    PloS one, 2011, Volume: 6, Issue:12

    Topics: 1-Deoxynojirimycin; Anesthetics; Animals; Chromatography, High Pressure Liquid; Chromatography, Liquid; Cornea; Cyclodextrins; Disease Progression; Drug Therapy, Combination; Enzyme Inhibitors; Genotype; Intracellular Signaling Peptides and Proteins; Lysosomes; Mass Spectrometry; Mice; Mice, Knockout; Microscopy, Confocal; Models, Biological; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Pregnanolone; Proteins

2011
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
    Journal of inherited metabolic disease, 2013, Volume: 36, Issue:1

    Topics: 1-Deoxynojirimycin; Child; Child, Preschool; Cognition; Deglutition; Disease Progression; Drug Administration Schedule; Female; Humans; Male; Nervous System Diseases; Niemann-Pick Disease, Type C; Time; Treatment Outcome

2013
Initiation and discontinuation of substrate inhibitor treatment in patients with Niemann-Pick type C disease.
    Gene, 2012, Sep-10, Volume: 506, Issue:1

    Topics: 1-Deoxynojirimycin; Adolescent; Carrier Proteins; Child; Child, Preschool; Developmental Disabilities; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Glucosyltransferases; Humans; Infant; Infant, Newborn; Intracellular Signaling Peptides and Proteins; Language Development Disorders; Male; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C

2012
N-butyldeoxynojirimycin inhibits murine melanoma cell ganglioside metabolism and delays tumor onset.
    Cancer letters, 2003, Nov-10, Volume: 201, Issue:1

    Topics: 1-Deoxynojirimycin; Animals; Cell Division; Cell Line, Tumor; Ceramides; Disease Progression; Dose-Response Relationship, Drug; Enzyme Inhibitors; Gangliosides; Glucosyltransferases; Melanoma, Experimental; Mice; Neoplasm Transplantation

2003
Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination.
    Epilepsia, 2007, Volume: 48, Issue:7

    Topics: 1-Deoxynojirimycin; Adult; Comorbidity; Disease Progression; Drug Administration Schedule; Drug Therapy, Combination; Dystonia; Electroencephalography; Enzyme Inhibitors; Epilepsies, Myoclonic; Gaucher Disease; Glucosylceramidase; Humans; Male; Nervous System Diseases; Recombinant Proteins; Syndrome; Treatment Outcome

2007