STAT+: Sarepta Therapeutics crisis is huge blow to Duchenne families, company

Sarepta Therapeutics confronted one of the most serious crises in its history on Friday, as the Food and Drug Administration prepared to ask the company to halt all shipments of its gene therapy for Duchenne muscular dystrophy — a treatment that had been a source of immense hope for patients and their families. 

A halt to shipments — following a series of patient deaths — would essentially remove the gene therapy, called Elevidys, from the market, at least temporarily. 

Two teenagers who were administered the gene therapy died earlier this year, while a 51-year-old man who received a similar Sarepta gene therapy in a clinical trial for a different form of muscular dystrophy also died. The third death occurred one month ago but only came to light on Thursday evening in media reports.

Continue to STAT+ to read the full story…