Clinical Trial Update: Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) [Phase 1, Phase 2]

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Summary

This clinical trial update reports completed Phase 1/2 studies evaluating pyrimethamine as a treatment for late-onset GM2 gangliosidosis, including Tay-Sachs an…

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Diseases / indications

• Gm2 Gangliosidosis
• Sandhoff Disease
• Tay-sachs Disease

Drugs mentioned

• Pyrimethamine (Daraprim) (Pyrimethamine)

Snippet

Sponsor: The Hospital for Sick Children | Conditions: Gangliosidoses, GM2, Sandhoff Disease | Phase 1, Phase 2 | Status: COMPLETED