Study wrapper · #992
Elamipretide in pediatric Barth syndrome: from heart failure to school return.
Amedro P, Andrianoely M, Gohier P, et al. Orphanet journal of rare diseases. 2026.
DOI: 10.1186/s13023-026-04487-zPubMed: 42415111
Editor's note
A single pediatric case report of elamipretide (SS-31) in Barth syndrome, with notable improvement alongside maximal standard therapy. As an uncontrolled n-of-1 report it cannot establish causation and one transient adverse event was noted. Carefully framed as a single-patient observation.
Plain-language abstract
In a single case report, a 5-year-old with Barth syndrome and severe heart failure showed improved heart function and quality of life over six months of elamipretide (SS-31).
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