Bakersfield man takes new drug to treat Friedreich's Ataxia

Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for the Treatment of Friedreich's Ataxia. A phase 2a investigator-initiated trial evaluating Elamipretide for the treatment of Friedreich's ataxia has been initiated at Children's Hospital of Philadelphia (CHOP) under the direction of Dr. David Lynch. The trial is evaluating two doses of Elamipretide in patients with Friedreich's ataxia to assess safety, visual function, and cardiac function. The Orphan Drug Act w

Stealth Bio's Elamipretide Gets Orphan Drug Designation From FDA For Friedreich's Ataxia

Help Develop New Treatments - National Ataxia Foundation

Columbia resident overcomes challenges of Friedreich's ataxia with newly-approved treatment