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The US Food and Drug Administration approved a Duchenne muscular dystrophy drug yesterday (December 13) that it had originally rejected in August, reports STAT. The approval is based on the results of a clinical trial that showed the drug increased the production of dystrophin, a protein that strengthens muscle fibers and is inhibited in people with Duchenne, a rare disorder that impedes muscle movement and can cause heart damage and death.
The drug, Vyondys 53 from Sarepta Therapeutics, is meant to treat a form of Duchenne caused by a genetic mutation in a sequence of the gene DMD called exon 53 that inhibits the production of dystrophin. About 8 percent of the 5,000 people in the US with Duchenne have this mutation. Vyondys 53 has been shown in a clinical trial to increase dystrophin production in people with Duchenne, but Sarepta has yet to show that ...
