New Option Approved for Inherited Metabolic Disease That Affects the Brain

(MedPage Today) — The FDA approved sepiapterin (Sephience) for the treatment of children and adults with phenylketonuria (PKU), PTC Therapeutics announced.
The approval includes broad labeling for the treatment of hyperphenylalaninemia in adult…

Source link : https://www.medpagetoday.com/genetics/generalgenetics/116718

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Publish date : 2025-07-29 16:33:00

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New Option Approved for Inherited Metabolic Disease That Affects the Brain

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