Podcast: Gene therapy could be best hope to cure Duchenne muscular dystrophy

, | | August 3, 2020
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Conner Curran, 9, and his brother Will, 7, playing on a swingset. Credit: Kholood Eid/NPR
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Conner Curran was 4 when he was diagnosed with Duchenne muscular dystrophy. His muscles were already beginning to waste away. A doctor offered Conner’s parents some advice.

JESSICA CURRAN: Take your son home. Love him. Take him on trips while he’s walking. Give him a good life and enjoy him because there’s really not many options right now.

SHAPIRO: Today, Conner is 9 and not just walking, but running. The reason – an experimental gene therapy.

HAMILTON: Gene therapy seemed like the answer. After all, children with Duchenne muscular dystrophy lack a functional version of a gene called dystrophin, which helps muscles stay healthy.

JUDE SAMULSKI: The concept is very simple. You’re missing a gene, so you’re putting it back.

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CONNER: I can run faster. I stand better, and I can walk to Goldberg’s. That’s a bagel shop. It’s more than two miles. And I couldn’t do that before.

HAMILTON: Conner’s body will never replace the muscle cells he lost, and it’s not clear how long his new genes will last. But the treatment seems to work. It’s now been tested on nine boys, and Pfizer is planning a much larger study for later this year. The therapy still has flaws, though. Several boys, including Conner, became ill temporarily after receiving the virus. Two ended up in the hospital. So Samulski’s been working on a fix.

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