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Clinical Trials for Sandhoff Disease

• A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
• N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)
• Synergistic Enteral Regimen for Treatment of the Gangliosidoses
• Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
• A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
• Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
• HSCT for High Risk Inherited Inborn Errors
• Stem Cell Transplant for Inborn Errors of Metabolism