Stealth BioTherapeutics is planning a Phase 2a clinical trial of elamipretide (SS-31) as a treatment for people with vision loss and/or heart disease associated with Friedreich’s ataxia (FA). The company expects to use data from this study — planned to start later this year — in designing a pivotal…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Six months of treatment with interferon-gamma, known as IFN-gamma, is safe and leads to significant improvements in walking skills and stance in people with Friedreich’s ataxia (FA), according to a small study in Turkey. However, no significant improvements were observed in other areas of motor coordination, such as sitting, upper…