Families Reeling After FDA Denies Access to Lifesaving Treatment

 

By Linda Childers

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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Source: NBC News