Study wrapper · #994
Elamipretide Improves Mitochondrial Function in Mitochondrial Trifunctional Protein-Deficient Mice and Human Fibroblasts.
Vieira Neto E, Wang M, Szuminsky AJ, et al. Journal of inherited metabolic disease. 2026.
DOI: 10.1002/jimd.70132PubMed: 42500837
Editor's note
A preclinical study testing elamipretide (SS-31) directly in a mouse model and human fibroblasts of trifunctional protein deficiency, reporting improved endurance and mitochondrial enzyme activity though cardiolipin content was unchanged. Framed as a candidate warranting further work, not an established therapy.
Plain-language abstract
In mice and patient-derived cells with a rare fatty-acid metabolism disorder, elamipretide (SS-31) was associated with improved mitochondrial function and exercise endurance.
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