Study wrapper · #991
Elamipretide: a mitochondria-targeting drug for Barth syndrome.
Villatore A, Ehle EA, Chelko SP Trends in pharmacological sciences. 2026.
DOI: 10.1016/j.tips.2026.06.011PubMed: 42448476
Editor's note
This review covers elamipretide (SS-31) for Barth syndrome, a use recently supported by regulatory approval. As a brief review it summarizes rather than generates evidence, but it centers a tracked peptide with a clear clinical narrative. Supports a measured, non-promotional writeup noting the ultra-rare indication.
Plain-language abstract
A short review discusses elamipretide (SS-31), a mitochondria-targeting peptide, in the context of Barth syndrome, a rare inherited mitochondrial disorder.
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