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elamipretide

Indications: - treatment of Barth syndrome Mechanism of action: - mitochondrial protective agent - theoretically improves the function of cardiolipin-deficient mitochondria

General

metabolic agent (metabolic modifier)

Database Correlations

PUBCHEM correlations

References

  1. 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