When Damon Woods of San Diego, Calif, was diagnosed four years ago
with Duchenne muscular dystrophy, a rare and fatal muscle disorder, his
mother, Charaine vowed to do whatever it took to save his life.
This week, Woods and other families were dealt a
blow when the Food and Drug Administration (FDA) declined to approve
Kyndrisa, generically known as drisapersen, an experimental drug that
had shown promise in treating Woods and other boys affected by Duchenne
muscular dystrophy.
According to the national non-profit, CureDuchenne,
Duchenne is the most common and lethal form of muscular dystrophy. As
the leading genetic killer of young boys, Duchenne affects more than
300,000 patients worldwide, primarily boys and young men, and there is
currently no FDA-approved therapy specifically designed to treat
Duchenne.
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