Results 151 to 160 of about 2,156 (206)

Sapropterin

Reactions Weekly
openaire   +2 more sources

Sapropterin/tozinameran

Reactions Weekly, 2023
openaire   +2 more sources

Sapropterin

Nature Reviews Drug Discovery, 2008
Barbara K Burton, Santwana Kar
exaly   +2 more sources

Sapropterin

Drugs, 2009
Sapropterin dihydrochloride (Kuvan), hereafter referred to as sapropterin, is a synthetic formulation of the active 6R-isomer of tetrahydrobiopterin, a naturally occurring cofactor for phenylalanine hydroxylase. In the EU, sapropterin is approved for the treatment of hyperphenylalaninaemia in patients >or=4 years of age with tetrahydrobiopterin ...
Mark Sanford
exaly   +3 more sources

Is Sapropterin Treatment Suitable for All Subjects With Phenylketonuria?

Expert Opinion on Pharmacotherapy, 2008
In a Phase I study, 20% of subjects with phenylketonuria (96/485) responded with a = 30% reduction in blood phenylalanine at day 8 of treatment with sapropterin. Subsequently, some of the responders from this study were enrolled in a Phase III study. The
Sheila A Doggrell
exaly   +1 more source

Safety and Efficacy of 22 Weeks of Treatment With Sapropterin Dihydrochloride in Patients With Phenylketonuria

American Journal of Medical Genetics, Part A, 2008
Phenylketonuria (PKU) is an inherited metabolic disease characterized by phenylalanine (Phe) accumulation, which can lead to neurocognitive and neuromotor impairment.
Marcello Giovannini, Udo Wendel, Jon Wolff   +2 more
exaly   +2 more sources

Spotlight on Sapropterin in Primary Hyperphenylalaninemia†

BioDrugs, 2009
Sapropterin dihydrochloride (Kuvan)) is a synthetic formulation of the active 6R-isomer of tetrahydrobiopterin, a naturally occurring co-factor for phenylalanine hydroxylase. In the EU, sapropterin is approved for the treatment of hyperphenylalaninemia in patients > or =4 years of age with tetrahydrobiopterin-responsive phenylketonuria (PKU), and in ...
Mark, Sanford, Gillian M, Keating
openaire   +2 more sources

Introduction of Sapropterin Dihydrochloride as Standard of Care in Patients With Phenylketonuria [PDF]

Molecular Genetics and Metabolism, 2010
Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been shown to reduce plasma phenylalanine (Phe) levels in a significant portion of patients with phenylketonuria (PKU). When we undertook introducing this medication to our PKU clinic population, the challenges of recalling and ...
Hilary J Vernon, Ada Hamosh, M R Johnson
exaly   +3 more sources

Efficacy and Safety of Sapropterin Dihydrochloride in Patients With Phenylketonuria: A Meta-Analysis of Randomized Controlled Trials

British Journal of Clinical Pharmacology, 2019
Aims The aim of the present meta‐analysis was to evaluate the efficacy and safety of sapropterin dihydrochloride in phenylketonuria (PKU) patients.
Yi-Min Cui, Jinghan Qu, Ying Zhou
exaly   +2 more sources

Effects of Sapropterin against Non-Alcoholic Fatty Liver Disease in mice

Immunological Investigations
Background Tetrahydrobiopterin (BH₄) maintains nitric oxide synthase (NOS) coupling. However, its therapeutic potential in non-alcoholic fatty liver disease (NAFLD) remains unexplored.
Jie Jiang, Zong-Qiang Hu, Li Li
semanticscholar   +1 more source