Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

Authors

• Fumihiko Urano, Washington University School of Medicine in St. Louis
• Bess A. Marshall, Washington University School of Medicine in St. Louis
• Stacy Hurst, Washington University School of Medicine in St. Louis
• Amy Robichaux-Viehoever, Washington University School of Medicine in St. Louis
• Saumel Ahmadi, Washington University School of Medicine in St. Louis
• Tamara Hershey, Washington University School of Medicine in St. Louis
• Gregory Van Stavern, Washington University School of Medicine in St. Louis
• Paulina Cruz Bravo, Washington University School of Medicine in St. Louis
• Jennifer Powers Carson, Washington University School of Medicine in St. Louis
• John Pesko, Amylyx Pharmaceuticals, Inc.
• Kelly Fox, Amylyx Pharmaceuticals, Inc.
• Nathalie Erpelding, Amylyx Pharmaceuticals, Inc.
• Camille L Bedrosian, Amylyx Pharmaceuticals, Inc.

Journal

Journal of Clinical Investigation

Publication Date

5-15-2026

Volume

136

Issue

10

First Page

e198519

Document Type

Open Access Publication

DOI

10.1172/JCI198519

Rights and Permissions

Urano F, Marshall BA, Hurst S, Robichaux-Viehoever A, Ahmadi S, Hershey T, Van Stavern G, Cruz Bravo P, Powers Carson J, Pesko J, Fox K, Erpelding N, Bedrosian CL. Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome. J Clin Invest. 2026 May 15;136(10):e198519. doi: 10.1172/JCI198519. © 2026, Urano et al. This is an open access article published under the terms of the Creative Commons Attribution 4.0 International License.

Recommended Citation

Urano, Fumihiko; Marshall, Bess A.; Hurst, Stacy; Robichaux-Viehoever, Amy; Ahmadi, Saumel; Hershey, Tamara; Van Stavern, Gregory; Cruz Bravo, Paulina; Powers Carson, Jennifer; Pesko, John; Fox, Kelly; Erpelding, Nathalie; and Bedrosian, Camille L, "Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome." Journal of Clinical Investigation. 136, 10. e198519 (2026).
https://digitalcommons.wustl.edu/oa_4/7735

Department

ICTS (Institute of Clinical and Translational Sciences)

Additional Links

Supplemental material is available for this article at publisher site.