Use of sapropterin in the management of phenylketonuria: Seven case reports

H Gokmen Ozel, A M Lammardo, K Motzfeldt, M Robert, J C Rocha, M van Rijn, K Ahring, A Bélanger-Quintana, A. Nicole MacDonald, K Dokoupil

5 Citationer (Scopus)

Abstract

Sapropterin treatment, with or without dietary treatment, improves blood phenylalanine control, increases phenylalanine tolerance, and may reduce the day-to-day dietary treatment burden in a subset of patients with phenylketonuria (PKU). Balancing the need for maintained control of blood phenylalanine with diet relaxation is complex when administering sapropterin. We present a series of seven patient cases with PKU that illustrate important aspects of using sapropterin with diet in the management of the disorder.
OriginalsprogEngelsk
TidsskriftMolecular Genetics and Metabolism
Vol/bind108
Udgave nummer2
Sider (fra-til)109-11
Antal sider3
ISSN1096-7192
DOI
StatusUdgivet - 2013

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