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.
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