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FDA issues approval for Duchenne Muscular Dystrophy gene therapy

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The approval of Sarepta Therapeutics’ gene therapy, Elevidys, is an ecstatic moment, but one laced with pain for a generation of parents.


This is a really good article on the impact of FDA approvals rare disease communities. The Spinal Muscular Atrophy community, my condition, got extremely lucky that 2 of the 3 treatments have no age or type restrictions. Even so seeing the treated kids, who would otherwise be dead, running upstairs, is amazing. There is a big fight in the SMA community regarding future advocacy, many including me, wanting parents to shift to social model disability issues such as Medicaid long term care and employment. It's a hard shift since big pharma money runs deep in these organizations. 


DMD is devastating, most of the people I know with it have a very "I'm going to die before 30 mindset" as it progresses. 

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