Jwheel86 Posted June 30, 2023 Share Posted June 30, 2023 Duchenne breakthrough therapy leaves behind pioneering families WWW.STATNEWS.COM 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. 1 1 2 Quote Link to comment Share on other sites More sharing options...
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