Rachel DeConti had been waiting for an announcement from Sarepta Therapeutics virtually every day since the hot summer afternoon she rushed her 4-year-old son Jacob to the hospital and learned his muscles were collapsing, spewing their contents into his bloodstream.
Just not this announcement.
“Hi,” the email said, “Please see the attached letter.” She felt sick as she read. Jacob, who was about to turn 9, would not receive the experimental gene therapy they had waited for. Nor would anyone else with his ultra-rare disease.
“They’ve been telling us for years, ‘it’s coming, it’s coming,’” said DeConti. “And then literally overnight, it’s gone.”
There has been no bigger story in biotechnology this summer than the saga of Sarepta and its gene therapy for Duchenne muscular dystrophy, a spectacle that has played volleyball with the hearts of parents, swung stock prices, and contributed to the brief ouster of a top Food and Drug Administration official.
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