In 2023, the U.S. Food and Drug Administration approved the first medication to treat people with Friedreich’s ataxia who are age 16 or older. Given that Friedreich’s ataxia is a genetic ...
has granted Orphan Drug Designation to Papillon’s PPL-001, an experimental treatment for Friedreich's ataxia. “We are pleased that the FDA has granted Orphan Drug Designation to our program ...