Gm2 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 · 2026-03-19Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
A Phase 2 clinical trial is evaluating the safety and efficacy of Nizubaglustat (AZ-3102) in patients with GM2 gangliosidosis and Niemann-Pi…
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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 · 2026-03-02Clinical Trial Update: A Natural History of Late Onset Tay-Sachs Disease
A completed natural history study of GM2 gangliosidosis, including late-onset Tay-Sachs disease, sponsored by Massachusetts General Hospital…
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CLINICAL_TRIALS trial_result · 2025-10-16Clinical Trial Update: Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
This clinical trial update reports long-term follow-up data for subjects treated with AXO-AAV-GM2 in GM2 gangliosidosis (Tay-Sachs and Sandh…
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CLINICAL_TRIALS trial_result · 2025-09-17Clinical Trial Update: Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) [Phase 3]
Phase 3 clinical trial evaluating miglustat therapy for infantile GM2 gangliosidosis (Sandhoff and Tay-Sachs diseases) has been terminated.
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CLINICAL_TRIALS research · 2025-03-20Clinical Trial Update: Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease [Phase 2]
Phase 2 study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics of oral AZ-3102 in GM2 gangliosidosis and Niemann-Pick …
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CLINICAL_TRIALS trial_result · 2024-03-12Clinical Trial Update: N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) [Phase 2]
Phase 2 clinical trial of N-Acetyl-L-Leucine for GM2 gangliosidosis (Tay-Sachs and Sandhoff) has completed with posted results.
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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.
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CLINICAL_TRIALS trial_result · 2016-05-19Clinical Trial Update: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis [Phase 2]
Phase 2 study of miglustat (Zavesca) in patients with GM2 gangliosidosis assessed pharmacokinetics and tolerability; results describe PK par…
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CLINICAL_TRIALS withdrawal · 2013-02-25Clinical Trial Update: A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis [Phase 1]
A Phase I clinical trial of pyrimethamine for GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases) has been withdrawn.
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CLINICAL_TRIALS trial_result · 2012-02-23Clinical Trial Update: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) [Phase 1, Phase 2]
This clinical trial update reports completed Phase 1/2 studies evaluating pyrimethamine as a treatment for late-onset GM2 gangliosidosis, in…
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CLINICAL_TRIALS trial_result · 2008-05-06Clinical Trial Update: Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses [Phase 3]
This Phase 3 trial update reports on the pharmacokinetics, safety and tolerability of miglustat (Zavesca) in infants with GM2 gangliosidosis…