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OALib Journal期刊
ISSN: 2333-9721
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-  2018 

Evidence- and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria

DOI: https://doi.org/10.1038/s41436-018-0403-z

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Abstract:

Phenylketonuria (PKU) is a rare metabolic disorder that requires life-long management to reduce phenylalanine (Phe) concentrations within the recommended range. The availability of pegvaliase (PALYNZIQ?, an enzyme that can metabolize Phe) as a new therapy necessitates the provision of guidance for its use

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