News

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…

Scientists have designed molecules to correct the effect of DNA expanded repeats that cause Friedreich’s ataxia (FA), according to a new study. Using patient-derived cells, experiments demonstrated that these molecules, known as anti-gene oligonucleotides, or A-GOs, markedly boosted the production of frataxin, which is the protein that’s deficient in…

Voyager Therapeutics expects to begin clinical testing of its experimental gene therapy for Friedreich’s ataxia (FA) in 2026. The company last year announced the selection of its FA gene therapy candidate, which is being developed in collaboration with Neurocrine Biosciences. At the time, Voyager had said…

Children with Friedreich’s ataxia are often underweight and short for their age, while adult patients are frequently overweight, so regular nutrition checks are important for maintaining healthy weight, a study from Europe suggested. The study, “Longitudinal analysis of anthropometric measures over 5 years in patients with Friedreich ataxia in…

Fundraisers, webinars, and social media campaigns are set for next month to help bring attention on Friedreich’s ataxia (FA), and provide support to those living with the disease. Leading those efforts is the Friedreich’s Ataxia Research Alliance (FARA), which is dedicated to raising funds and aligning efforts for…

Using MRI scans, a team of researchers identified several differences in the brain and spinal cord of people with Friedreich’s ataxia (FA) versus those without the rare genetic condition, which have the potential to serve as biomarkers of disease progression in clinical trials. The findings come from TRACK-FA…

The investigational gene therapy LX2006 was well tolerated and led to clinically meaningful improvements in cardiac biomarkers and functional measures in people with Friedreich’s ataxia (FA) and a type of heart disease called cardiomyopathy, the results from two clinical trials show. The findings informed the design of a…

Larimar Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) this year to grant accelerated approval to nomlabofusp, its experimental treatment for Friedreich’s ataxia (FA). “The strong clinical and regulatory progress across our nomlabofusp program reinforces the timing of our planned Biologics License Application (BLA)…

CAP-004, Capsida Biotherapeutics’ experimental gene therapy for Friedreich’s ataxia (FA), safely delivered its genetic cargo to key disease-related tissues in nonhuman primates, new research shows. Specifically, that cargo was delivered to the heart, sensory nerves, and the central nervous system, or CNS, which comprises the brain and spinal…

Biogen’s Skyclarys (omaveloxolone) has been approved in Canada to treat people with Friedreich’s ataxia (FA) ages 16 and older, becoming the first disease-modifying therapy for FA to receive regulatory clearance in the country. The therapy was approved under Health Canada’s priority review process, which accelerates the review timeline…