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What happens when a $3m gene therapy is not enough

LOS ANGELES - Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to walk, talk, swallow or even breathe. So when in 2021 his parents heard about Zolgensma – a one-time therapy costing millions of dollars that.

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What happens when a $2 million gene therapy is not enough?

What happens when a $2 million gene therapy is not enough?
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Healthy Kids: New hope for kids with Duchenne muscular dystrophy after FDA grants gene therapy approval

Healthy Kids: New hope for kids with Duchenne muscular dystrophy after FDA grants gene therapy approval
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Nationwide Children's Hospital pioneers new gene therapy for Duchenne muscular dystrophy

In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children's, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children's Hospital, where the therapy was invented and initially tested.

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Curing the incurable: FDA makes history, approves Elevidys to treat pediatric Duchenne muscular dystrophy

The U.S. Food and Drug Administration approved Elevidys in June, making it the first gene therapy designed to treat pediatric Duchenne muscular dystrophy, a life-threatening genetic disorder of progressive muscular weakness.

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