Time to Functional Loss as an Endpoint in Huntington's Disease Trials: Enrichment and Sample Size.
James A MillsJeffrey D LongJatin G VaidyaEmily C GantmanSwati SatheSarah J TabriziCristina SampaioPublished in: Movement disorders : official journal of the Movement Disorder Society (2024)
PIN is predictive of functional decline. A delay of 9 months or more in the transition to Stage 3 for an enriched sample yields feasible sample size estimates, demonstrating that this approach can aid in planning future trials. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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