Early trial tests drug to save sight in rare neurological disease

This small, completed pilot study tested whether a drug called elamipretide is safe and might help control vision loss in people with advanced Friedreich ataxia (FRDA). Twenty participants with FRDA-related vision or heart problems received either a low or high dose of the drug for up to a year to see if it could improve their eyesight or other symptoms. The main goal was to check safety and see if the drug could help people read more letters on an eye chart after treatment.