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 trial_result · 2025-10-24Clinical Trial Update: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
This clinical trial update reports that the natural history study of GM1 Gangliosidosis in infantile and juvenile patients has completed. It…
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CLINICAL_TRIALS trial_result · 2025-09-29Clinical Trial Update: GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
A clinical trial update for the PROspective Neurological Disease Trajectory Study (PRONTO) by Azafaros reports completion of the GM1 and GM2…
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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…
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CLINICAL_TRIALS trial_result · 2021-04-14Clinical Trial Update: Synergistic Enteral Regimen for Treatment of the Gangliosidoses [Phase 4]
Phase 4 trial update for a synergistic enteral regimen in GM1 and GM2 gangliosidoses; the study was terminated with results posted in 2021.