Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

Fumihiko Urano; Bess 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

doi:10.1172/JCI198519

Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

Fumihiko Urano
, Bess 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

Research output: Contribution to journal › Article › peer-review

Abstract

PB&TURSO was associated with improved or stabilized pancreatic function, vision, and overall symptom burden in individuals with Wolfram syndrome, a rare and progressive degenerative disease.

Original language	English
Journal	The Journal of clinical investigation
Volume	136
Issue number	10
DOIs	https://doi.org/10.1172/JCI198519
State	Published - May 15 2026

Keywords

Cell stress
Clinical trials
Endocrinology
Genetic diseases
Genetics

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10.1172/JCI198519

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@article{749e65e93cd04b3d9fa862d402652735,

title = "Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome",

abstract = "PB\&TURSO was associated with improved or stabilized pancreatic function, vision, and overall symptom burden in individuals with Wolfram syndrome, a rare and progressive degenerative disease.",

keywords = "Cell stress, Clinical trials, Endocrinology, Genetic diseases, Genetics",

author = "Fumihiko Urano and Bess Marshall and Stacy Hurst and Amy Robichaux-Viehoever and Saumel Ahmadi and Tamara Hershey and \{Van Stavern\}, Gregory and \{Cruz Bravo\}, Paulina and \{Powers Carson\}, Jennifer and John Pesko and Kelly Fox and Nathalie Erpelding and Bedrosian, \{Camille L.\}",

year = "2026",

month = may,

day = "15",

doi = "10.1172/JCI198519",

language = "English",

volume = "136",

journal = "The Journal of clinical investigation",

issn = "0021-9738",

number = "10",

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T1 - Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

AU - Urano, Fumihiko

AU - Marshall, Bess

AU - Hurst, Stacy

AU - Robichaux-Viehoever, Amy

AU - Ahmadi, Saumel

AU - Hershey, Tamara

AU - Van Stavern, Gregory

AU - Cruz Bravo, Paulina

AU - Powers Carson, Jennifer

AU - Pesko, John

AU - Fox, Kelly

AU - Erpelding, Nathalie

AU - Bedrosian, Camille L.

PY - 2026/5/15

Y1 - 2026/5/15

N2 - PB&TURSO was associated with improved or stabilized pancreatic function, vision, and overall symptom burden in individuals with Wolfram syndrome, a rare and progressive degenerative disease.

AB - PB&TURSO was associated with improved or stabilized pancreatic function, vision, and overall symptom burden in individuals with Wolfram syndrome, a rare and progressive degenerative disease.

KW - Cell stress

KW - Clinical trials

KW - Endocrinology

KW - Genetic diseases

KW - Genetics

UR - https://www.scopus.com/pages/publications/105039335802

U2 - 10.1172/JCI198519

DO - 10.1172/JCI198519

M3 - Article

C2 - 42138079

AN - SCOPUS:105039335802

SN - 0021-9738

VL - 136

JO - The Journal of clinical investigation

JF - The Journal of clinical investigation

IS - 10

ER -

Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome

Abstract

Keywords

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