Title of article
Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study
Author/Authors
Harvey L Levy، نويسنده , , Andrzej Milanowski، نويسنده , , Anupam Chakrapani، نويسنده , , Maureen Cleary، نويسنده , , Philip Lee، نويسنده , , Friedrich K Trefz، نويسنده , , Chester B Whitley، نويسنده , , François Feillet، نويسنده , , Annette S Feigenbaum، نويسنده , , Judith D Bebchuk، نويسنده , , Heidi Christ-Schmidt، نويسنده , , Alex Dorenbaum، نويسنده ,
Issue Information
روزنامه با شماره پیاپی سال 2008
Pages
7
From page
504
To page
510
Abstract
Background
Early and strict dietary management of phenylketonuria is the only option to prevent mental retardation. We aimed to test the efficacy of sapropterin, a synthetic form of tetrahydrobiopterin (BH4), for reduction of blood phenylalanine concentra
Journal title
The Lancet
Serial Year
2008
Journal title
The Lancet
Record number
591753
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