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◆ Journal of Huntington s Disease2025-11-26· Clinical trial

Huntington's disease clinical trials update: October 2025

Mena Farag, Sarah J. Tabrizi, Edward J. Wild

原始摘要(英文原文)· Original abstract
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the launch of the phase II/III clinical trial of SKY-0515 from Skyhawk Therapeutics and the phase I/II clinical trial of SPK-10001 from Spark Therapeutics. We also report positive topline data from uniQure's phase I/II clinical trial of AMT-130 after 36 months of follow-up. Further updates include recent developments in Roche's tominersen programme within GENERATION HD2, progress with votoplam (PTC518) in PIVOT-HD by PTC Therapeutics and developments in the collaborative PTC Therapeutics/Novartis programme. We additionally discuss regulatory developments regarding pridopidine following the negative PROOF-HD study. Finally, we provide an updated listing of all registered and ongoing clinical trials in Huntington's disease.
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Huntington's disease clinical trials update: October 2025 — 科研速览 Science Skim