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Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: Results of a phase 3b study

✍ Scribed by Barbara K. Burton; Maria Nowacka; Julia B. Hennermann; Mark Lipson; Dorothy K. Grange; Anupam Chakrapani; Friedrich Trefz; Alex Dorenbaum; Michael Imperiale; Sun Sook Kim; Paul M. Fernhoff

Book ID: 116989324
Publisher: Elsevier Science
Year: 2011
Tongue: English
Weight: 290 KB
Volume: 103
Category: Article
ISSN: 1096-7192
DOI: 10.1016/j.ymgme.2011.03.020

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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 FDA‐approved synthetic formulation of tetrahydrobiopterin (6R‐BH4, herein referred to as

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