Barth Syndrome is a genetic disease so rare that fewer than 200 Americans have it, and there are under 300 known cases worldwide. It hinders the mitochondria in cells and primarily impairs the heart’s functionality. Americans with the illness were given a ray of hope in recent years thanks to an experimental drug called Elamipretide. The FDA recently refused a request to reconsider a drug application from its makers, prolonging the federal approval process.
Author: Inside Edition
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News post in August 24, 2025, 6:05 am.
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