Sarepta Therapeutics announced it is pausing shipments of its Duchenne muscular dystrophy gene therapy for patients who cannot walk, following the death of a second individual who received the treatment. The first death, reported in March, involved a 16-year-old boy. Both fatalities were attributed to acute liver failure, a known side effect of some gene therapies. The affected patients were non-ambulatory, meaning their condition had advanced to the stage where they required wheelchairs. Most children with Duchenne lose the ability to walk by their teenage years. Sarepta is collaborating with experts to develop an improved immunosuppressive regimen to enhance the safety of the therapy, named Elevidys, particularly for non-ambulatory patients. The company plans to consult with the Food and Drug Administration regarding this proposed regimen.
— new from statnews.com
