Time to Functional Loss as an Endpoint in Huntington's Disease Trials: Enrichment and Sample Size
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.
Authors
- James A. Mills (ORCID: https://orcid.org/0009-0000-5762-7566)
- Cristina Sampaio (ORCID: https://orcid.org/0000-0002-7052-9079)
- Jatin G. Vaidya (ORCID: https://orcid.org/0000-0002-2375-816X)
- Swati Sathe (ORCID: https://orcid.org/0000-0002-9972-955X)
- Jeffrey D. Long (ORCID: https://orcid.org/0000-0001-7181-9652)
- Sarah J. Tabrizi (ORCID: https://orcid.org/0000-0003-2716-2045)
- Emily C. Gantman (ORCID: https://orcid.org/0000-0002-6527-7559)
Institutions
- University of Iowa (US)
- CHDI Foundation (US)
- UK Dementia Research Institute (GB)
- National Hospital for Neurology and Neurosurgery (GB)
- University College London (GB)
Publication Details
- Journal
- Movement Disorders
- Published
- 2024-08-05
- DOI
- https://doi.org/10.1002/mds.29963
- Citations
- 1
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.27
Funders
- CHDI Foundation