Elamipretide

How it works

Grips cardiolipin, a lipid of the inner membrane of mitochondria, the cell's power plants. For muscle weakness in Barth syndrome.

What it's made of

4 synthetic amino acids (D-Arg-Dmt-Lys-Phe): one in “mirror” form and another modified. It binds to cardiolipin, a lipid of the inner membrane of the mitochondrion.

What it's for

Prescribed for

Improving muscle strength in Barth syndrome, a rare mitochondrial disease (mostly in males), in patients weighing 30 kg or more.