Sapropterin Is Safe and Effective in Patients less than 4-Years-Old with BH4 -Responsive Phenylalanine Hydroxylase Deficiency

Objective To assess the safety and efficacy of tetrahydrobiopterin therapy with sapropterin to treat tetrahydrobiopterin (BH4 )-responsive phenylalanine hydroxylase (PAH) deficiency in children aged

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Bibliographic Details
Published in:The Journal of pediatrics 2014-12, Vol.165 (6), p.1241-1244
Main Authors: Shintaku, Haruo, MD, PhD, Ohura, Toshihiro, MD, PhD
Format: Article
Language:English
Subjects:
Pediatrics
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Description
Summary:Objective To assess the safety and efficacy of tetrahydrobiopterin therapy with sapropterin to treat tetrahydrobiopterin (BH4 )-responsive phenylalanine hydroxylase (PAH) deficiency in children aged
ISSN:0022-3476
1097-6833
DOI:10.1016/j.jpeds.2014.08.003