Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models

Guoqiao Wang; Zhaolong Adrian Li; Ling Chen; Heather Lugar; Tamara Hershey

doi:10.1371/journal.pone.0321598

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Open Access

Peer-reviewed

Research Article

Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models

Guoqiao Wang ,

Roles Conceptualization, Data curation, Formal analysis, Methodology, Writing – original draft, Writing – review & editing

* E-mail: guoqiao@wustl.edu, tammy@wustl.edu

Affiliations Department of Neurology, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America, Division of Biostatistics, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America

https://orcid.org/0000-0002-6808-8737

⨯
Zhaolong Adrian Li,

Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

Affiliation Department of Psychiatry, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America

https://orcid.org/0000-0003-2246-4116

⨯
Ling Chen,

Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

Affiliation Division of Biostatistics, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America
⨯
Heather Lugar,

Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

Affiliations Department of Neurology, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America, Department of Psychiatry, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America
⨯
Tamara Hershey

Roles Conceptualization, Methodology, Supervision, Writing – original draft, Writing – review & editing

Affiliations Department of Neurology, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America, Department of Psychiatry, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America, Department of Radiology, Washington University in St Louis School of Medicine, St Louis, Missouri, United States of America
⨯

Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models

Guoqiao Wang,
Zhaolong Adrian Li,
Ling Chen,
Heather Lugar,
Tamara Hershey

Published: May 9, 2025
https://doi.org/10.1371/journal.pone.0321598
See the preprint

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