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Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome
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
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
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Research Letter Endocrinology Genetics

Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

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Abstract

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

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Figure 1

Clinical and functional outcomes.

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Clinical and functional outcomes.
(A) Mean and participant-level trough-...
(A) Mean and participant-level trough-adjusted C-peptide AUC at 120 minutes into 240-minute MMTTs at baseline and weeks 24 and 48. All participants improved (green dots), except 1 per-protocol and 2 ITT participants (black dots). Asterisk denotes overlapping data points (both green). The y axis scales differ between graphs due to single ITT outlier. (B) Mean C-peptide levels during MMTTs at baseline and weeks 24 and 48 (95% CIs are provided in the Supporting Data Values file). (C and D) Summary and participant-level data for secondary glycemic control measures: HbA1c (C) and time in target glucose range (D; 70–180 mg/dL). (E) Best-corrected visual acuity (BCVA) as assessed by logarithm of the minimum angle of resolution (LogMAR) in the best eye. (F) Symptom burden at weeks 24 and 48 assessed via PGI-C and CGI-C. All measured participants were responders (defined by ratings of minimally, much, or very much improved or no change). Means (bars), 95% CIs (error bars), and mean changes (brackets) are shown in A and C–E. In participant-level data, green indicates improved or stable, and black indicates others. Single asterisks denote overlapping points of same color; double asterisks denote 1 green and 1 black.

Copyright © 2026 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)

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