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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…

The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, MDA vice president of disability outreach and empowerment. The Muscular Dystrophy Association (MDA) is once again hosting its annual gathering, the MDA Clinical & Scientific Conference. The 2025 event will take place…

The U.S. Food and Drug Administration (FDA) granted priority review to PTC Therapeutics‘ application seeking the approval of vatiquinone, an oral treatment for children and adults with Friedreich’s ataxia (FA). The agency will announce a decision by Aug. 19, the company said. “We are excited to be…

Skyclarys (omaveloxolone) improves heart function in Friedreich’s ataxia (FA) by reducing abnormal heartbeats and stabilizing heart rhythm, a study in a mouse disease model of severe cardiomyopathy concluded. Data also revealed sex-specific disturbance in the heart’s electrical activity and responses to Skyclarys treatment in these mice. The study,…

People with Friedreich’s ataxia (FA) were found in a new study to experience more mental health symptoms — particularly decreased motivation and greater anxiety and depression — than individuals without the rare disease, who served as controls. The results of this survey study, conducted in the Czech Republic, also…

Two microRNAs (miRNAs) — tiny molecules that control the activity of genes — are associated with structural brain and heart damage in Friedreich’s ataxia (FA) patients, according to a study from Brazil. FA patients were found to have higher levels of one miRNA, and lower levels of…

Adolescents with Friedreich’s ataxia (FA) are now being dosed with nomlabofusp as part of a placebo-controlled Phase 1 clinical study that’s testing Larimar Therapeutics‘ subcutaneous, or under-the-skin, injection therapy. Data from the Phase 1 study, and from other clinical testing of nomlabofusp, are expected by the middle…

Errors during repair when DNA gets damaged may give rise to the mutations that cause Friedreich’s ataxia, a study revealed. The study, “Recurrent DNA nicks drive massive expansions of (GAA)n repeats,” was published in PNAS. A cell’s DNA stores the instructions to make the proteins and…

The U.S. Food and Drug Administration (FDA) has given Solid Biosciences the go-ahead to start clinical testing of SGT-212, the company’s novel gene therapy candidate for Friedreich’s ataxia (FA). According to Solid, SGT-212 is the only experimental gene therapy of its kind to target “both neurologic and…