Page last updated: 2024-08-23

miglustat and Adult GM1 Gangliosidosis

miglustat has been researched along with Adult GM1 Gangliosidosis in 7 studies

Research

Studies (7)

Timeframe	Studies, this research(%)	All Research%
pre-1990	0 (0.00)	18.7374
1990's	0 (0.00)	18.2507
2000's	3 (42.86)	29.6817
2010's	3 (42.86)	24.3611
2020's	1 (14.29)	2.80

Authors

Authors	Studies
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, R	1
Di Rocco, M; Favia, V; Fischetto, R; Gaeta, A; Giacomini, T; Giordano, P; Lassandro, G; Mancardi, MM; Morrone, A; Palladino, S; Palladino, V; Romano, A; Severino, M; Strisciuglio, P; Tripaldi, ME; Zampini, L	1
Jarnes Utz, JR; Kim, S; King, K; Redtree, ES; Schema, L; Whitley, CB; Ziegler, R	1
Caciotti, A; Deodato, F; Dionisi-Vici, C; Donati, MA; Morrone, A; Procopio, E; Rampazzo, A; Scarpa, M; Taurisano, R	1
Butters, TD; d'Azzo, A; Dwek, RA; Elliot-Smith, E; Jeyakumar, M; Perry, VH; Platt, FM; Smith, DA; Thomas, R; van der Spoel, AC	1
Brady, RO	1
Butters, TD; d'Azzo, A; Dwek, RA; Elliot-Smith, E; Jeyakumar, M; Lloyd-Evans, E; Platt, FM; Smith, DA; Speak, AO; van der Spoel, AC	1

Reviews

1 review(s) available for miglustat and Adult GM1 Gangliosidosis

Article	Year
Emerging strategies for the treatment of hereditary metabolic storage disorders. Rejuvenation research, 2006,Summer, Volume: 9, Issue:2 Topics: 1-Deoxynojirimycin; Bone Marrow Transplantation; Enzyme Inhibitors; Fabry Disease; Gangliosidosis, GM1; Gaucher Disease; Genetic Therapy; Humans; Imino Sugars; Piperidines	2006

Trials

1 trial(s) available for miglustat and Adult GM1 Gangliosidosis

Article	Year
Infantile gangliosidoses: Mapping a timeline of clinical changes. Molecular genetics and metabolism, 2017, Volume: 121, Issue:2 Topics: 1-Deoxynojirimycin; Diet, Ketogenic; Disaccharidases; Female; Gangliosidoses; Gangliosidoses, GM2; Gangliosidosis, GM1; Glycoside Hydrolase Inhibitors; Humans; Infant; Male; Prospective Studies; Retrospective Studies	2017

Other Studies

5 other study(ies) available for miglustat and Adult GM1 Gangliosidosis

Article	Year
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
Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience. Molecular genetics & genomic medicine, 2020, Volume: 8, Issue:10 Topics: 1-Deoxynojirimycin; Adolescent; Central Nervous System; Child; Child, Preschool; Drug Tolerance; Female; Gangliosidosis, GM1; Glucosyltransferases; Glycoside Hydrolase Inhibitors; Humans; Infant; Male	2020
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
Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis. Brain : a journal of neurology, 2003, Volume: 126, Issue:Pt 4 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD; Apoptosis; Biomarkers; Blood-Brain Barrier; Cytokines; Enzyme Inhibitors; Gangliosidoses; Gangliosidoses, GM2; Gangliosidosis, GM1; Genes, MHC Class II; Immunohistochemistry; Inflammation; Mice; Sandhoff Disease; Tay-Sachs Disease	2003
Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis. Molecular genetics and metabolism, 2008, Volume: 94, Issue:2 Topics: 1-Deoxynojirimycin; Animals; beta-Galactosidase; Brain; Disease Models, Animal; Enzyme Inhibitors; Feces; Gangliosidosis, GM1; Glucosyltransferases; Glycosphingolipids; Humans; Macrophage Activation; Mice; Mice, Knockout; Motor Activity; Protein Transport	2008