Sandhoff Disease
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CLINICAL_TRIALS research · 2026-03-06Clinical Trial Update: Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis
A translational update on ex vivo gene therapy approaches for GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) with status not yet recru…
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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 trial_result · 2026-01-28Clinical Trial Update: A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 [Phase 3]
Phase 3 multinational trial of venglustat for late-onset GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) was terminated; results have b…
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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 · 2025-09-29Clinical 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 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 · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS trial_result · 2025-05-22Clinical Trial Update: A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease [Phase 1]
Phase 1 dose-escalation and safety/efficacy study of AXO-AAV-GM2 for Tay-Sachs Disease and Sandhoff Disease was terminated, with results pos…
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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 · 2014-12-04Clinical Trial Update: Gene Therapy for Tay-Sachs Disease
This clinical trial update indicates that a University of Minnesota-sponsored gene therapy study for Tay-Sachs disease, Sandhoff disease, an…
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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…