(USNEWS) — WASHINGTON (AP) — Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication.
The approval comes nearly five months after the Food and Drug Administration and a panel of outside advisers panned the drug, saying there was little evidence that it helped. But regulators faced a public backlash from patients' families, politicians and physicians.
The FDA cleared Sarepta Therapeutics' Exondys 51 for a rare form of Duchenne muscular dystrophy, a deadly inherited disease that affects boys. It's the first FDA approval for the degenerative condition, which causes muscle weakness, loss of movement and eventually death.
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