Gm1 Gangliosidosis
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis [Phase 3]
A Phase 3 trial is evaluating oral Nizubaglustat (AZ-3102) for late-infantile and juvenile GM1 gangliosidosis and GM2 gangliosidosis, and th…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis [Phase 3]
This clinical trial update notes that oral Nizubaglustat (AZ-3102) is being studied in a Phase 3 trial for late-infantile and juvenile Niema…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: A Natural History Study of the Gangliosidoses
This clinical trial update describes an ongoing natural history study of gangliosidoses. It includes Tay-Sachs disease, Sandhoff disease, GM…
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CLINICAL_TRIALS research · 2025-12-11Clinical Trial Update: A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis [Phase 1, Phase 2]
An ongoing Phase 1/2 clinical trial is evaluating intravenous delivery of an AAV9 vector expressing human beta-galactosidase in patients wit…
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
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CLINICAL_TRIALS research · 2025-05-21Clinical Trial Update: Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis [Phase 1, Phase 2]
Gemma Biotherapeutics reports an ongoing Phase 1/2 trial evaluating PBGM01 for safety, tolerability, and efficacy in pediatric GM1 gangliosi…