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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…

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…