Patients and advocates in the rare disease community are celebrating the approval of elamipretide, a medication that some feared would become unavailable due to its long regulatory approval process.

The approval “represents a historic breakthrough for the Barth Syndrome community and for every family affected by mitochondrial disease,” said the Plum-based United Mitochondrial Disease Foundation in a statement. “For decades, families have been told there is little that can be done. Now, for the first time, we have an FDA-approved therapy that addresses the root cause of a mitochondrial disease.”

The medicine stabilizes mitochondrial membranes to improve the function of mitochondria, the energy-producing powerhouses of a cell. When they do not function correctly, the body may lack the ener

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