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Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias

โœ Scribed by Blau, Nenad


Book ID
121745546
Publisher
Informa plc
Year
2013
Tongue
English
Weight
492 KB
Volume
9
Category
Article
ISSN
1742-5255

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## Abstract Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to neurocognitive and neuromotor impairment. Sapropterin dihydrochloride, an FDAโ€approved synthetic formulation of tetrahydrobiopterin (6Rโ€BH4, herein referred to as