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Sapropterin dihydrochloride

National Institutes of Health

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Breast Cancer Resistance Protein Inhibitors [MoA]Sapropterin dihydrochloride 100 MG Oral Tablet [Kuvan]Sapropterin dihydrochloride 500 MG Powder for Oral Solution [Kuvan]

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Papers overview

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Review
2019
Review
2019
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria.
2015
2015
Long-term safety and efficacy of sapropterin: the PKUDOS registry experience.
Review
2013
Review
2013
Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias
Introduction: Phenylketonuria (PKU) is caused by mutation of the enzyme, phenylalanine (Phe) hydroxylase (PAH). The… 
Highly Cited
2013
Highly Cited
2013
Long-term Follow-up and Outcome of Phenylketonuria Patients on Sapropterin: A Retrospective Study
OBJECTIVE: Sapropterin dihydrochloride, the synthetic form of 6R-tetrahydrobiopterin (BH4), is an approved drug for the treatment… 
2012
2012
Recommendations for the use of sapropterin in phenylketonuria.
2012
2012
Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria
BackgroundSapropterin dihydrochloride, an EMEA-approved synthetic formulation of BH4, has been available in Europe since 2009 for… 
Review
2011
Review
2011
Diagnosis, classification, and genetics of phenylketonuria and tetrahydrobiopterin (BH4) deficiencies.
2011
2011
Safety of sapropterin dihydrochloride (6r–bh4) in patients with pulmonary hypertension
ABSTRACT The authors investigated the safety of oral tetrahydrobiopterin (BH4), a cofactor for nitric oxide synthesis, as a novel… 
Highly Cited
2008
Highly Cited
2008
Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria
Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to… 
Highly Cited
2007
Highly Cited
2007
Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study
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