Tay-Sachs Disease
Pipeline by Development Stage
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (3)
Total enrollment: 151 patients across 3 trials
Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
A Natural History Study of the Gangliosidoses
Phase Legend
Key Insights
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