elamipretide
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Indications
- treatment of Barth syndrome
Mechanism of action
- mitochondrial protective agent
- theoretically improves the function of cardiolipin-deficient mitochondria
More general terms
References
- ↑ Lou N Elamipretide for Barth Syndrome Survives FDA Advisory Vote. Panelists acknowledge difficulty generating stronger data in ultra-rare disease. MedPage Today October 11, 2024 https://www.medpagetoday.com/publichealthpolicy/fdageneral/112353