Tag: elamipretide
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A Race Against Time: Families Hope for FDA Approval of Life-Changing Drug for Barth Syndrome
A Race Against Time: Families Hope for FDA Approval of Life-Changing Drug for Barth Syndrome Desperate parents petition the FDA as a promising treatment for a devastating rare disease faces regulatory hurdles. For families grappling with Barth syndrome, a relentless genetic disorder that disproportionately affects young boys, the passage of time is a stark and…