STAT+: For Duchenne moms who pushed for cures, new breakthrough therapy can’t rebuild what’s lost
The approval of Sarepta Therapeutics’ gene therapy, Elevidys, is an ecstatic moment, but one laced with pain for a generation of parents.
Pat Furlong was sitting in her home office in Middletown, Ohio, last Thursday, refreshing a Food and Drug Administration web page ad nauseam, when the phone rang. She answered and burst into tears.
The FDA had just approved the first gene therapy for Duchenne muscular dystrophy, her friend and the therapy’s architect, Jerry Mendell, told her. It was a culmination of advocacy work Furlong began 39 years prior, after her own sons were diagnosed with the fatal muscle-wasting disease.
She thought back to them, Christopher and Patrick, and trips they had taken in 1992 to Memphis, where researchers were experimenting with implanting immature muscle cells into Duchenne patients. She was full of hope then. One of that year’s radio hits was Marc Cohn’s “Walking in Memphis,” and Furlong kept hearing the song’s central line in her head: You’ve got a prayer in Memphis.
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