AAV gene therapy to treat Friedreich’s ataxia cardiomyopathy

Ferreira, J. AAV gene therapy to treat Friedreich’s ataxia cardiomyopathy. Lab Anim 53, 86 (2024). doi:10.1038/s41684-024-01351-0

NeuroVoices: Francesco Saccà, MD, PhD, on Crossing Over Dimethyl Fumarate in Friedriech Ataxia

NeurologyLive. April 3, 2024. We completed the enrollment in February. All 40 patients were enrolled. We are expecting to have the last patient last visit by the end of July. This means that we could probably get some data, at least the primary and many of the secondary endpoints, by September or October of this year. By the end of the year, we will close the entire analysis.

Approval of omaveloxolone for Friedreich ataxia

Boesch, S., Indelicato, E. Approval of omaveloxolone for Friedreich ataxia. Nat Rev Neurol (2024). doi:10.1038/s41582-024-00957-9 

The recent approval of omaveloxolone for the treatment of Friedreich ataxia in the USA and Europe represents an important milestone in the field of rare neurological diseases. However, many challenges lie ahead, including the translation of trial results into clinical practice, and the management of patients’ expectations.