Muscular dystrophy gene therapy nears approval, but safety concerns linger
Five years ago, when Duchenne muscular dystrophy (DMD) began making it hard for him to walk, 7-year-old Conner Curran received a blood infusion of trillions of viruses carrying a muscle gene to replace his mutant one. Within 2 months the Connecticut boy went from crawling up stairs to “flying up,” says his mother, Jessica Curran. The family and the researchers hoped he would never need another gene infusion. But the experimental treatment’s effects are already fading. Conner’s story sums up the mixture of hope and disappointment among families and researchers as gene therapy for DMD, long seen as a potential cure for the debilitating and ultimately fatal disease, reaches a key milestone. This week, the U.S. Food and Drug Administration (FDA) was expected to approve a treatment similar to Conner’s, developed by Sarepta Therapeutics. ...