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Sapropterin dihydrochloride

National Institutes of Health

Papers overview

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Review
2013
Review
2013
  • N. Blau
  • Expert opinion on drug metabolism & toxicology
  • 2013
  • Corpus ID: 36872881
Introduction: Phenylketonuria (PKU) is caused by mutation of the enzyme, phenylalanine (Phe) hydroxylase (PAH). The… Expand
2012
2012
Phenylketonuria (PKU) is an inherited disorder of phenylalanine (Phe) metabolism. Until recently, the only treatment for PKU was… Expand
  • table 1
2012
2012
BackgroundSapropterin dihydrochloride, an EMEA-approved synthetic formulation of BH4, has been available in Europe since 2009 for… Expand
Review
2011
Review
2011
This article summarizes the present knowledge, recent developments, and common pitfalls in the diagnosis, classification, and… Expand
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  • table 1
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2010
2010
Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been… Expand
  • figure 2
  • table 1
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  • table 2
Highly Cited
2009
Highly Cited
2009
OBJECTIVE To evaluate the ability of sapropterin dihydrochloride (pharmaceutical preparation of tetrahydrobiopterin) to increase… Expand
Review
2009
Review
2009
Phenylketonuria (PKU) is caused by mutations in the phenylalanine hydroxylase (PAH) gene, leading to deficient conversion of… Expand
  • table 1
  • figure 2
  • figure 1
  • figure 3
  • figure 4
Highly Cited
2008
Highly Cited
2008
Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to… Expand
Highly Cited
2007
Highly Cited
2007
BACKGROUND Early and strict dietary management of phenylketonuria is the only option to prevent mental retardation. We aimed to… Expand
Highly Cited
2007
Highly Cited
2007
SummaryThis study aimed to evaluate the response to and safety of an 8-day course of sapropterin dihydrochloride (6R… Expand