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Wolfram syndrome and a phase II trial of sodium phenylbutyrate and taurursodiol
In this episode, Fumihiko Urano describes how the manuscript reports 24- and 48-week results from the ongoing HELIOS trial (ClinicalTrials.gov NCT05676034), a single-center, single-arm, open-label, phase II trial of PB&TURSO. Collectively, these results support continued development of PB&TURSO.
Published May 15, 2026, by JCI Staff
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Authors
Fumihiko Urano, Bess A. Marshall, Stacy Hurst, Amy Robichaux-Viehoever, Saumel Ahmadi, Tamara Hershey, Gregory Van Stavern, Paulina Cruz Bravo, Jennifer Powers Carson, John Pesko, Kelly Fox, Nathalie Erpelding, Camille L. Bedrosian
Copyright © 2026 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)