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Sapropterin dihydrochloride

 
National Institutes of Health

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Highly Cited
2013
Highly Cited
2013
OBJECTIVE: Sapropterin dihydrochloride, the synthetic form of 6R-tetrahydrobiopterin (BH4), is an approved drug for the treatment… Expand
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2012
2012
Phenylketonuria (PKU) is an inherited disorder of phenylalanine (Phe) metabolism. Until recently, the only treatment for PKU was… Expand
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2012
2012
BackgroundSapropterin dihydrochloride, an EMEA-approved synthetic formulation of BH4, has been available in Europe since 2009 for… Expand
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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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Highly Cited
2009
Highly Cited
2009
OBJECTIVE To evaluate the ability of sapropterin dihydrochloride (pharmaceutical preparation of tetrahydrobiopterin) to increase… Expand
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Review
2009
Review
2009
Phenylketonuria (PKU) is caused by mutations in the phenylalanine hydroxylase (PAH) gene, leading to deficient conversion of… Expand
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Highly Cited
2008
Highly Cited
2008
Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to… Expand
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2008
2008
AbstractBackground and objective: Untreated phenylketonuria is characterized by neurocognitive and neuromotor impairment, which… Expand
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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
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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
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