How the Moms and Dads of Patients Won Sarepta's Drug a Reprieve
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The parents leaped into action as soon as health officials asked Sarepta Therapeutics Inc. to stop selling an expensive gene therapy used to treat a rare muscle disease that affects boys and young men.
Some traveled to Washington to meet with members of Congress. Others reached out to White House contacts. Still others wrote letters to the Food and Drug Administration directly. They demanded the FDA restore access to the drug Elevidys for Duchenne muscular dystrophy — even though a big trial failed to prove it slowed the disease's progression. In fact, for some, the treatment appeared to be deadly.
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