The deadly saga of the controversial gene therapy Elevidys
In the days since, the company has acquiesced. But its reputation has already been hit. And the events have dealt a devastating blow to people desperate for treatments that might help them, their children, or other family members with DMD. DMD is a rare genetic disorder that causes muscles to degenerate over time. It’s caused by a mutation in a gene that codes for a protein called dystrophin. That protein is essential for muscles—without it, muscles weaken and waste away. The disease mostly aff