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FDA Approves First Treatment for Rare Barth Syndrome

1 min read
Source: fda.gov
FDA Approves First Treatment for Rare Barth Syndrome
Photo: fda.gov
TL;DR

The FDA has granted accelerated approval to Forzinity (elamipretide) as the first treatment for Barth syndrome, a rare and serious mitochondrial disease, based on improvements in muscle strength that are likely to benefit patients, with further studies required to confirm these benefits.

Topics:businesshealth#barth-syndrome#elamipretide#fda#forzinity#health#rare-diseases
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  1. FDA Grants Accelerated Approval to First Treatment for Barth Syndrome  fda.gov
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  3. FDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards  statnews.com
  4. Orland Park baby's treatment for rare genetic disease Barth Syndrome granted accelerated approval by Food and Drug Administration  ABC7 Chicago
  5. A rare disease exhausted him. A drug gives him energy to fight for others.  AJC.com

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