Story thread · 4 reports / 4 sources

My son’s disease progressed while the FDA waited

conservativereview.com · 10dShare

Birthdays should mark milestones, offer hope for another year, and remind us to cherish the people we love. But on the first anniversary of the Food and Drug Administration’s decision to restrict access to Elevidys, a gene therapy for Duchenne muscular dystrophy, there will be no cake or candles in our home. The only birthdays Duchenne families want to celebrate are the ones our children are still here to have. The FDA should help us light those candles. My 14-year-old son, Ryu, is still fighting to reach another birthday. A year ago, I was exploring Elevidys as a treatment that might help him live a longer, fuller life. Then, almost overnight, that hope disappeared. The FDA first halted use of the therapy entirely before restoring access only for boys who could still walk. Ryu, who already uses a wheelchair, was left behind. By age 14, more than 82% of Duchenne patients use a wheelchair. After age 15, the share exceeds 90%. I have spent my life living with Duchenne muscular dystrophy

First report: FDA panel rejects Duchenne therapy over efficacy concerns — beckershospitalreview.com, 12d