We use cookies to help provide you with the best possible online experience. Please read our Privacy Policy for information about which cookies we use and what information we collect on our site. By continuing to use this site, you agree that we may store and access cookies on your device.

I Agree

You are here: Industry Insights > Barth Syndrome

News List

Stealth BioTherapeutics Receives Orphan Drug Designation from the European Medicines Agency for Elamipretide for the Treatment of Barth Syndrome firstwordpharma
June 02, 2021
MITO today announced that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.
FDA Grants Rare Pediatric Disease Designation to Elamipretide for Barth Syndrome americanpharmaceuticalreview
March 05, 2020
Stealth BioTherapeutics announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for elamipretide for the treatment of Barth syndrome.
Stealth BioTherapeutics Announces Positive Results for Elamipretide in Barth Syndrome Open-Label Extension Trial americanpharmaceuticalreview
April 18, 2019
Stealth BioTherapeutics announced positive results from the open-label extension of the Phase 2/3 TAZPOWER study of elamipretide ...