Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria-Reference-Cited by-同舟云学术

Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria

Published:2008-11-15 Issue:22 Volume:146A Page:2851-2859
ISSN:1552-4825
Container-title:American Journal of Medical Genetics Part A
language:en
Short-container-title:Am. J. Med. Genet.

Author:

Lee Phillip,Treacy Eileen P.,Crombez Eric,Wasserstein Melissa,Waber Lewis,Wolff Jon,Wendel Udo,Dorenbaum Alex,Bebchuk Judith,Christ-Schmidt Heidi,Seashore Margretta,Giovannini Marcello,Burton Barbara K.,Morris Andrew A.,

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Reference32 articles.

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3. Spanish BH4-responsive phenylalanine hydroxylase-deficient patients: Evolution of seven patients on long-term treatment with tetrahydrobiopterin;Belanger-Quintana;Mol Genet Metab,2005

4. Social outcome in treated individuals with inherited metabolic disorders: UK study;Bhat;J Inherit Metab Dis,2005

5. The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): A phase II, multicentre, open-label, screening study;Burton;J Inherit Metab Dis,2007

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