Andrea Lobo,  —

Skyclarys (omaveloxolone) may improve the function of peripheral sensory nerves — those found outside the brain and spinal cord, which detect sensations and help provide the brain with information — in people with Friedreich’s ataxia (FA). That’s according to a new report from Slovenia describing the cases of two sisters…

A newly awarded $300,000 research grant will fund a study on heart fibrosis, or the accumulation of scar tissue, in people with Friedreich’s ataxia (FA). The Muscular Dystrophy Association (MDA) and Friedreich’s Ataxia Research Alliance (FARA) awarded the grant to researchers at Weill Cornell Medical Center and Indiana…

The volume of gray matter in the brain region known as the cerebellum helps predict motor and cognitive benefits for Friedreich’s ataxia patients after noninvasive neuromodulation, a therapy that delivers mild electric currents to brain regions, a study found. Gray matter contains a high concentration of nerve cell bodies…

The approved Friedreich’s ataxia (FA) drug Skyclarys (omaveloxolone) was found to be generally safe and well tolerated in people with FA in the real world, according to a new U.S. study that tested the medication’s safety over one year. Most patients remained on Skyclarys for more than 12 months…

A study of heart health with vatiquinone, an investigational oral Friedreich’s ataxia (FA) therapy, found no increased risk of cardiac problems in healthy adults, including when doses about 3.5 times above the therapeutic dose were evaluated, scientists reported. Blood levels of vatiquinone did not correlate with changes in…

The U.S. Food and Drug Administration (FDA) has given rare pediatric disease designation to PPL-001, Papillon Therapeutics’ investigational cell therapy for Friedreich’s ataxia (FA). This status is granted to potential medicines or therapy approaches meant to treat serious or life-threatening diseases affecting fewer than 200,000 people in…

People with Friedreich’s ataxia (FA) may have elevated blood levels of cardiac troponin I and T, markers of heart damage, without signs of heart dysfunction, a study suggests. Being younger, female, or having a higher number of GAA repeats, the main type of FA-causing mutation, was associated with higher…

The U.S. Food and Drug Administration (FDA) has removed the partial hold on the clinical development of nomlabofusp, an experimental treatment for Friedreich’s ataxia (FA). The decision followed a review of data from a Phase 2 dose exploration trial (NCT05579691), which showed the treatment was well tolerated…

The U.S. Food and Drug Administration (FDA) has put Lexeo Therapeutics’ LX2006, a gene therapy candidate for heart disease in people with Friedreich’s ataxia (FA), on the fast track for development. The award of fast track status aims to expedite the development and regulatory review of investigational therapies…

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…

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 (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…