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Ultragenyx's gene therapy gets FDA OK for glycogen storage disorder
FDA grants Ultragenyx's Genglycos accelerated approval, its first gene therapy, for GSDIa in patients 8 and up; list price $2.7M.
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healio.com
FDA approves first gene therapy to treat glycogen storage disease type Ia
The FDA has granted accelerated approval of a gene therapy to treat children and adults aged at least 8 years with glycogen storage disease type Ia, according to an industry press…
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fiercepharma.com
Ultragenyx, after FDA speed bump, lands 1st gene therapy nod with green light in glycogen storage disease
If at first you don’t succeed in gene therapy, then try, try again. | If at first you don’t succeed in gene therapy, then try, try again, is the lesson Ultragenyx likely took home…
