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.
Use of sapropterin in the management of phenylketonuria : seven case reports / H. Ozel Gokmen, A.M. Lammardo, K. Motzfeldt, M. Robert, J.C. Rocha, M. van Rijn, K. Ahring, A. Bélanger Quintana, A. Macdonald, K. Dokoupil. - In: MOLECULAR GENETICS AND METABOLISM. - ISSN 1096-7192. - 108:2(2013 Feb), pp. 109-111. [10.1016/j.ymgme.2012.11.012]
Use of sapropterin in the management of phenylketonuria : seven case reports
A.M. LammardoSecondo
;
2013
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.File | Dimensione | Formato | |
---|---|---|---|
1-s2.0-S1096719212004416-main.pdf
accesso riservato
Tipologia:
Publisher's version/PDF
Dimensione
135.33 kB
Formato
Adobe PDF
|
135.33 kB | Adobe PDF | Visualizza/Apri Richiedi una copia |
Pubblicazioni consigliate
I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.