News

Top 10 Friedreich’s ataxia stories of 2023

Friedreich’s Ataxia News consistently covered the latest in scientific research, treatment development, and clinical studies for Friedreich’s ataxia (FA) throughout 2023. Here are the top 10 most widely read stories of last year: No. 10 – Dosing starts in 2nd patient group in trial of gene therapy candidate…

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…

Intensive rehabilitation seen to benefit adults and children with FA

An intensive and multidisciplinary rehabilitation program eased symptoms of ataxia — lack of coordination and muscle control — and improved motor function in adults and children with Friedreich’s ataxia (FA), a study reported. The sweeping program offered physiotherapy, occupational therapy, manual activities, psychological support, speech therapy, and clinical psychology.

New MRI technique captures changes in FA brains

Noninvasive diffusion MRI (dMRI) captured changes in the structure of the brain and brainstem of people with Friedreich’s ataxia (FA), a study reports. dMRI, a new method that relies on the flow of water in tissue to assess neurodegeneration, has the potential to identify therapeutic targets in FA patients,…

FA treatments that target mitochondria may best help patients

Therapies that aid mitochondria, cellular organelles responsible for producing most of the energy needed by cells, may help with physical function and heart health in people with Friedreich’s ataxia (FA), according to a review of published studies. Findings appear to support the benefits of Skyclarys (omaveloxolone), the only…