Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models
For rare diseases like Wolfram syndrome, leveraging existing resources such as historical/external controls and run-in data, along with evaluating comprehensive treatment effects using bivariate/multivariate endpoints, can significantly expedite the development of new drugs.
Authors
- Heather M. Lugar (ORCID: https://orcid.org/0000-0002-7917-5545)
- Tamara Hershey (ORCID: https://orcid.org/0000-0001-7549-0698)
- Guoqiao Wang
- Ling Chen (ORCID: https://orcid.org/0000-0003-0238-1831)
- Zhaolong Li (ORCID: https://orcid.org/0000-0003-2246-4116)
Institutions
- Washington University in St. Louis (US)
Publication Details
- Journal
- PLoS ONE
- Published
- 2025-05-09
- DOI
- https://doi.org/10.1371/journal.pone.0321598
- Primary Topic
- Folate and B Vitamins Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- American Diabetes Association
- National Institutes of Health