An Fda Pathway Can Accelerate Innovation for Duchenne Muscular Dystrophy


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"There's no choice between watching your child die young or pursuing a potentially revolutionary treatment," writes a mother whose 6-year-old son has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, guaranteed death by early adulthood from cardiac or respiratory failure.

"For parents of boys with Duchenne, dystrophin is our white whale, the life-or-death imperative no parenting manual can prepare you for."

So when her son qualified for a clinical trial of a gene therapy that introduces a smaller, functional form of the dystrophin gene, she and her husband "did not think twice."

The gene therapy, called eteplirsen, was approved by the FDA in 2016 and has since been shown to increase life span by about 20%, about five years longer than the average Duchenne patient.

"It demonstrates that a small amount of supplemental dystrophinjust 0.9% with eteplirsencan meaningfully lengthen lives," writes Erin Gloria Ryan in an op-ed for the Washington Post.

"Families know what those added years represent: more celebrations, more laughs, more stories."

But she also writes about the "arbitrary restrictions on approval of this gene therapy" that are keeping many families from

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