Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study-Reference-Cited by-同舟云学术

Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study

Published:2007-08 Issue:9586 Volume:370 Page:504-510
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Levy Harvey L,Milanowski Andrzej,Chakrapani Anupam,Cleary Maureen,Lee Philip,Trefz Friedrich K,Whitley Chester B,Feillet François,Feigenbaum Annette S,Bebchuk Judith D,Christ-Schmidt Heidi,Dorenbaum Alex

Publisher

Elsevier BV

Subject

General Medicine

Reference38 articles.

1. Phenylketonuria: epitome of human biochemical genetics (second of two parts);Scriver;N Engl J Med,1980

2. Phenylketonuria: epitome of human biochemical genetics (first of two parts);Scriver;N Engl J Med,1980

3. Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. Chapter 77;Donlon,2004

4. Phenylketonuria in adulthood: a collaborative study;Koch;J Inherit Metab Dis,2002

5. Blood phenylalanine control in adolescents with phenylketonuria;Walter;Int J Adolesc Med Health,2004

Cited by 255 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data;Molecular Genetics and Metabolism;2024-03

2. Maximal dietary responsiveness after tetrahydrobiopterin (BH4) in 19 phenylalanine hydroxylase deficiency patients: What super-responders can expect;Molecular Genetics and Metabolism Reports;2024-03

3. The treatment of biochemical genetic diseases: From substrate reduction to nucleic acid therapies;Molecular Genetics and Metabolism;2023-11

4. Expert Consensus on the Long-Term Effectiveness of Medical Nutrition Therapy and Its Impact on the Outcomes of Adults with Phenylketonuria;Nutrients;2023-09-11

5. Neuropsychiatric Function Improvement in Pediatric Patients with Phenylketonuria;The Journal of Pediatrics;2023-09