Phase II trial of sodium phenylbutyrate and taurursodiol in Wolfram syndrome.
other · Level IV
Where this comes from
- Record sourced from PubMed, PMID 42138079.
- Also identified by DOI 10.1172/JCI198519 and PMC identifier 13178640.
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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.
Medical subject headings
- Wolfram Syndrome
- Phenylbutyrates