Evidence map›Paper›PMID 42138079›Full record

ArticleThe Journal of clinical investigation2026

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 and 3 more

Abstract read
In one paragraph

Article in The Journal of clinical investigation, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Fumihiko UranoDepartment of Medicine.
Bess A MarshallDepartments of Pediatrics and Cell Biology & Physiology.
Stacy HurstDepartment of Medicine.
Amy Robichaux-ViehoeverDepartment of Neurology.
Saumel AhmadiDepartment of Neurology.
Tamara HersheyDepartments of Psychiatry and Radiology, and.
Gregory Van StavernDepartment of Ophthalmology & Visual Sciences, Washington University School of Medicine, St. Louis, Missouri, USA.
Paulina Cruz BravoDepartment of Medicine.
Jennifer Powers CarsonDepartment of Medicine.
John PeskoAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
Kelly FoxAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
Nathalie ErpeldingAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
Camille L BedrosianAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.

Funding

WU P&FP30DK020579 · NIDDK · WASHINGTON UNIVERSITY · PI Clay F. Semenkovich · 2013 to 2026
$27.1M
WUIDDRC Supplement-Supporting the health and well-being of children with intellectual and developmental disability during COVID-19 pandemicP50HD103525 · NICHD · WASHINGTON UNIVERSITY · PI JOSEPH D DOUGHERTY, Natasha Marrus · 2020 to 2026
$15.5M
Molecular Genetics of Hereditary Endoplasmic Reticulum DiabetesR01DK132090 · NIDDK · WASHINGTON UNIVERSITY · PI JANGA, SARATH CHANDRA, URANO, FUMIHIKO · 2022 to 2025
$2.2M
NICHD NIH HHS P50 HD103525NIDDK NIH HHS P30 DK020579NIDDK NIH HHS R01 DK132090
6 · The paper itself

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.

Indexed as

PhenylbutyratesWolfram SyndromeDrug CombinationsHumansTaurochenodeoxycholic Acid4-phenylbutyric acidDrug CombinationsPhenylbutyratessodium phenylbutyrate and taurursodiolTaurochenodeoxycholic AcidCell stressClinical trialsEndocrinologyGenetic diseasesGenetics

Identifiers

PMID42138079
PMCPMC13178640

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.