Results 101 to 110 of about 6,013 (134)
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 ...
Javier de las Heras +2 more
exaly +9 more sources
Sapropterin Dihydrochloride for the Treatment of Hyperphenylalaninemias
Expert Opinion on Drug Metabolism and Toxicology, 2013 Phenylketonuria (PKU) is caused by mutation of the enzyme, phenylalanine (Phe) hydroxylase (PAH). The hyperphenylalaninemia characteristic of PKU causes devastating neurological damage if not identified and treated at birth with a Phe-restricted diet.
Nenad Blau
exaly +5 more sources
Characterization of Sapropterin Dihydrochloride Tablet [PDF]
Journal of Drug Delivery and TherapeuticsCharacterization of Kuvan tablets involved thorough analysis of physical, chemical, and dissolution properties. Formulation development encompassed pre-formulation studies, feasibility trials, and process optimization. Kuvan tablets demonstrated variability in sapropterin dihydrochloride concentration but rapid dissolution across different media ...
Rakesh Kumar Jat, Tapan Kumar Jena
exaly +3 more sources