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Description:
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In this episode we turn the spotlight on a rare but debilitating disease affecting young boys, Duchenne muscular dystrophy.
Duchenne boys have a genetic mutation that affects their ability to produce a protein essential for normal muscle function. By their pre-teen years, boys with Duchenne are almost always confined to wheelchairs. They are only expected to live into their 20s. And despite decades of research and telethon fundraising, there is still no cure.
But two companies have developed drugs to treat Duchenne and are currently racing to get them approved by the Food and Drug Administration.
But while the government deliberates, the countdown clock for boys with Duchenne is ticking.
Illustration by Molly Ferguson for STAT
Music:
Time to Move and Motivate, by The Insider
Snowing, by Peter Rudenko
Our Ego [feat. Different Visitor], by Broke For Free
Gloci, by Graham Bole
Elefly, by Eino Toivanen |
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