Federal health regulators have granted tentative approval to a highly contested drug for muscular dystrophy that has become a flashpoint in the debate over patient access to experimental medicine.
The Food and Drug Administration said it cleared Sarepta Therapeutics' injection 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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Monday's announcement comes nearly five months after the agency and a panel of outside advisers panned the drug at a public meeting, saying there was little evidence of its effectiveness. But regulators faced an immense outcry from patient groups and physicians seeking access to the drug.