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UPDATE (Nov. 25):

An FDA panel reviewed a drug for Duchenne muscular dystrophy called drisaperson on Tuesday. What it found is not encouraging. The Signal podcast takes a look at what happened and where things go from here.


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 called dystrophin, which is 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.


Into this hopelessness step two companies: Sarepta Therapeutics and BioMarin Pharmaceutical. Both have developed drugs to treat Duchenne and are currently racing to get them approved by the Food and Drug Administration. But the path to get there hasn’t been easy — each has had their own corporate saga. Company bigwigs were ousted, stocks soared and tanked, partnerships were created and dissolved. What once looked like promising data has dwindled to a questionable body of evidence for both these drugs. As these dramas play out, parents of boys with Duchenne are waiting. And their kids are only getting worse.

But the wait, and the drama, may be over soon. On Nov. 24, an outside panel of FDA advisors is meeting to discuss whether BioMarin’s drug should be approved. It’s unclear if the drug will meet the rigorous standards set by the FDA to balance safety concerns against the potential benefit of a drug.


Max, left, 13, and Austin Leclaire, 16, outside of their apartment in Pembroke, Mass.. Both boys have Duchenne muscular dystrophy, and are taking an experimental drug. Kayana Szymczak for STAT

But given the amount of suffering these little boys go through, should the FDA approve them, anyway? How much evidence is enough? And as companies start to tackle drugs for rare diseases, is this is a dilemma the FDA is going to be seeing more of?

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The Signal podcast is produced by Katie Hiler.


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