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Storia in 3 fonti
First Therapy Approved for Dangerous Metabolic Disorder
Patients with GSDIa need to constantly eat cornstarch to avoid dangerous drops in blood sugar
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3 prospettive sulla stessa storiaTimeline cronologica
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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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endpoints.news
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.
