An intravenous FXN gene therapy was generally well tolerated in 17 patients with Friedreich ataxia cardiomyopathy and showed early signs of reducing heart damage. Further investigation is still needed. doi.org/hb736g
A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal inherited cardiac disease, according to a phase 1 clinical trial led by Weill Cornell Medicine researchers.
