PTC Therapeutics reports positive results for Huntington's drug votoplam

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PTC Therapeutics Reports Positive Topline Results from Month 24 Interim Analysis of PIVOT-HD Extension Study of Votoplam

April 28, 2026 4:05 PM EDT

- Dose-dependent benefit on cUHDRS in Stage 2 participants compared to matched natural history cohort, with 52% slowing at 10 mg dose -

- Continued evidence of favorable safety profile - 

- Novartis initiated global Phase 3 INVEST-HD study of votoplam -

- PTC will host a conference call to discuss results today, April 28, at 4:30 pm ET - 

WARREN, N.J., April 28, 2026 /PRNewswire/... More