Investigational Gene Therapy AVXS-401 Shows Promise in Animal Models of FA

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GENE THERAPY FOR FRIEDEICH’S ATAXIA: AVXS-401 , an experimental gene therapy for Friedreich’s ataxia (FA), is safe, well-tolerated, and leads to clinically meaningful improvements in mice and primate models of the disease, early studies have found. According to researchers, these promising findings suggest that AVXS-401 is safe for human use and support beginning clinical […]