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Could Desperate Parents’ Pleas Sway The FDA To Approve A Drug Even If Evidence It Works Is Lacking?

Shared by Radhika Narayanan | 104 34 0 | about 3 years ago

You don’t usually hear people compare Food and Drug Administration advisory committee meetings to a world-class sporting event, but this is how Brian Denger described the one set for Monday:

“For the Duchenne community, this is going to be more like the Super Bowl,” Denger, a supermarket manager from Biddeford, Maine, told me.

The “Duchenne community” refers to people touched by Duchenne muscular dystrophy, or DMD, the most common and most severe form of muscular dystrophy. Denger is a member because he is the father of two sons with DMD.

Worldwide, about 1 in 3,500 boys–DMD almost exclusively affects boys–inherit the genetic disease, which is passed on the “X” chromosome from mothers to sons. Affected boys begin to exhibit symptoms of muscle weakness between the ages of 3 and 5. By 12, most can no longer walk. Eventually, the disease weakens their heart and the muscles involved in breathing, and they die, typically before their 30th birthday.

No approved drug can slow the course of any type of muscular dystrophy, let alone reverse or cure it, which is why hundreds of members of the Duchenne community–parents, patients, doctors, scientists and other advocates–are expected Monday to pack the Chesapeake Ballroom at the College Park Marriott Hotel and Conference Center in the Washington, D.C., suburb of Hyattsville, Md., a few miles from the FDA’s sprawling Silver Spring campus.

Read More On www.forbes.com

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