Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study (Q84286469)

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3 February 2020

title

Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study (English)

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sapropterin dihydrochloride

3 February 2020

main subject

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ChEBI release 2020-05-28

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ChEBI release 2020-05-28

5

Dorothy K Grange

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3 February 2020

author name string

Barbara K Burton

1

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3 February 2020

Maria Nowacka

2

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3 February 2020

Julia B Hennermann

3

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Mark Lipson

4

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Anupam Chakrapani

6

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3 February 2020

Friedrich Trefz

7

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Alex Dorenbaum

8

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Michael Imperiale

9

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Sun Sook Kim

10

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Paul M Fernhoff

11

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publication date

31 March 2011

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Molecular Genetics and Metabolism

3 February 2020

published in

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volume

103

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issue

4

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page(s)

315-322

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Meta-analysis of neuropsychological symptoms of adolescents and adults with PKU.

3 February 2020

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2011.03.020

The reality of dietary compliance in the management of phenylketonuria

7 January 2021

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Tetrahydrobiopterin-responsive phenylalanine hydroxylase deficiency

7 January 2021

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Tetrahydrobiopterin as an alternative treatment for mild phenylketonuria

7 January 2021

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Long-term treatment and diagnosis of tetrahydrobiopterin-responsive hyperphenylalaninemia with a mutant phenylalanine hydroxylase gene

7 January 2021

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Successful treatment of phenylketonuria with tetrahydrobiopterin

7 January 2021

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Efficiency of long-term tetrahydrobiopterin monotherapy in phenylketonuria

7 January 2021

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Long-term treatment with tetrahydrobiopterin increases phenylalanine tolerance in children with severe phenotype of phenylketonuria

7 January 2021

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Spanish BH4-responsive phenylalanine hydroxylase-deficient patients: evolution of seven patients on long-term treatment with tetrahydrobiopterin

7 January 2021

1 reference

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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

7 January 2021

1 reference

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

7 January 2021

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

7 January 2021

1 reference

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Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study

7 January 2021

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Outcome and long-term follow-up of 36 patients with tetrahydrobiopterin deficiency

7 January 2021

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Recommendations on the dietary management of phenylketonuria. Report of Medical Research Council Working Party on Phenylketonuria

7 January 2021

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Plasma phenylalanine in patients with phenylketonuria self-managing their diet

7 January 2021

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Rationale for the German recommendations for phenylalanine level control in phenylketonuria 1997.

7 January 2021

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Management of phenylketonuria and hyperphenylalaninemia: the French guidelines

7 January 2021

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10.1016/J.YMGME.2011.03.020

7 January 2021

Identifiers

DOI

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3 February 2020

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