Andrea Lobo,  —

EU committee favors approval of Skyclarys for FA in Europe

The Committee for Medicinal Products for Human Use (CHMP) — an arm of the European Medicines Agency — is recommending the approval of Skyclarys (omaveloxolone) for treating Friedreich’s ataxia (FA) in people ages 16 and older in the European Union. The European Commission now will review the CHMP’s…

Dosing beings in 2nd patient group in Phase 1/2 trial of gene therapy

A first group of patients has been treated with LX2006, an investigational gene therapy for heart disease in people with Friedreich’s ataxia (FA), in a Phase 1/2 clinical trial and dosing has begun in a second group. LX2006 has been well tolerated and not associated with unexpected…

Short-read genome sequencing test may help diagnose atypical FA

Short-read genome sequencing (SR-GS), a test that can identify difficult-to-detect mutations, may help to correctly diagnose atypical Friedreich’s ataxia (FA) in people whose symptoms and family history are not indicative of FA, a study in Germany shows. The study, “Short-read genome sequencing allows ‘en route’ diagnosis of patients…