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Spotlight on sapropterin in primary hyperphenylalaninemia

Mark Sanford1, Gillian M Keating

  • 1Wolters Kluwer Health mid R: Adis, Auckland, New Zealand, an editorial office of Wolters Kluwer Health, Philadelphia, Pennsylvania, USA.

Biodrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
|July 25, 2009
PubMed
Summary

Sapropterin dihydrochloride is a novel treatment for phenylketonuria (PKU). This synthetic tetrahydrobiopterin formulation effectively lowers blood phenylalanine levels in responsive PKU patients.

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