The story of Sarepta's Duchenne gene therapy

The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The fate of Sarpeta’s gene therapy for the disease, called Elevidys, has been the center of a dizzying saga that seems to change by the minute. The deaths of two older boys have been tied to the treatment, and the company has gone back and forth with the Food and Drug Administration over which patients can access Elevidys. The development and approval of Elevidys was not without controversy, either. It was approved despite big questions about its efficacy, but many families of patients who have been treated with the drug report that their children are seeing positive results. In this latest installment of STATus Report, senior multimedia producer Alex Hogan breaks down the story of Elevidys. Starting from when it was first approved, all the way up to Tuesday night’s breaking news that Vinay Prasad, the official who had been overseeing Sarepta’s gene therapy at FDA, is out at the agency. Alex also chats with Adam Feuerstein, STAT’s senior biotech writer, about the past week’s whiplash-inducing news, and we’ll hear from a mother whose son has Duchenne. More from STAT: Become a subscriber: https://www.statnews.com/stat-plus/ Watch Full Episodes: https://www.youtube.com/@Statnews Newsletters: https://www.statnews.com/signup/ STAT events: https://www.statnews.com/stat-events/ Facebook: https://www.facebook.com/statnews Twitter: https://twitter.com/statnews Instagram: https://www.instagram.com/statnews/ Flipboard: rb.gy/3xnsxr STAT Reports: https://rb.gy/rexfwj STAT eBooks: https://rb.gy/eme3h5 ABOUT STAT: