News

Good safety seen with FA drug Skyclarys over 1 year in real world

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 design molecules that target genetic defects in FA

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…

Nutrition monitoring may help patients keep healthy weight: Study

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…

Larimar to seek nomlabofusp’s accelerated approval this year

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)…

MDA 2025: Experimental gene therapy boosts frataxin in primates

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…

Friedreich’s ataxia treatment Skyclarys approved in Canada

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…