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Sapropterin dihydrochloride for phenylketonuria and tetrahydrobiopterin deficiency
Expert Review of Endocrinology and Metabolism, 2010 Sapropterin dihydrochloride is the first registered synthetic form of the naturally occurring cofactor and cosubstrate, tetrahydrobiopterin (BH4). It is essential for the conversion of phenylalanine (Phe) by phenylalanine-4-hydroxylase (PAH) to tyrosine.
Nenad Blau
exaly +4 more sources
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria [PDF]
Molecular Genetics and Metabolism, 2019 Phenylketonuria (PKU) is an inherited metabolic disease caused by phenylalanine hydroxylase (PAH) deficiency. As the resulting high blood phenylalanine (Phe) concentration can have detrimental effects on brain development and function, international guidelines recommend lifelong control of blood Phe concentration with dietary and/or medical therapy ...
Ania C Muntau +2 more
exaly +8 more sources
American Journal of Medical Genetics, Part A, 2008 AbstractPhenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to neurocognitive and neuromotor impairment. Sapropterin dihydrochloride, an FDA‐approved synthetic formulation of tetrahydrobiopterin (6R‐BH4, herein referred to as sapropterin) is effective in reducing plasma Phe ...
Jon Wolff +2 more
exaly +4 more sources
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Sapropterin Dihydrochloride Tablets
Hospital Pharmacy, 2008Each month, subscribers to The Formulary Monograph Service receive 5 to 6 well-documented monographs on drugs that are newly released or are in late phase 3 trials. The monographs are targeted to your Pharmacy & Therapeutics Committee. Subscribers also receive monthly 1-page summary monographs on the agents that are useful for agendas and pharmacy ...
Dennis J. Cada +2 more
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PP14 Budget Impact Of Sapropterin Dihydrochloride For Phenylketonuria
International Journal of Technology Assessment in Health Care, 2019IntroductionThe National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system.
Eduardo Mulinari +2 more
openaire +1 more source