Results 151 to 160 of about 1,551 (187)

Systematic literature review of the somatic comorbidities experienced by adults with phenylketonuria. [PDF]

open access: yesOrphanet J Rare Dis
Whitehall KB   +17 more
europepmc   +1 more source

Sapropterin Dihydrochloride Tablets

Hospital Pharmacy, 2008
Each 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
openaire   +1 more source

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

Pharmacokinetics of Sapropterin in Patients with Phenylketonuria

Clinical Pharmacokinetics, 2008
Untreated phenylketonuria is characterized by neurocognitive and neuromotor impairment, which result from elevated blood phenylalanine concentrations. To date, the recommended management of phenylketonuria has been the use of a protein-restricted diet and the inclusion of phenylalanine-free protein supplements; however, this approach is often ...
F. Feillet   +11 more
openaire   +3 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, Gillian M, Keating
openaire   +2 more sources

Sapropterin: A New Therapeutic Agent for Phenylketonuria

Annals of Pharmacotherapy, 2009
Objective: To summarize the role of pharmacotherapy in the management of phenylketonuria (PKU) and to review the pharmacology, pharmacokinetics, pharmacodynamics, efficacy data, and safety profile of sapropterin for this indication. Data Sources: A literature search was conducted using MEDLINE (1966–May 2009), International Pharmaceutical Abstracts ...
Karly A, Hegge   +3 more
openaire   +2 more sources

Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias

Expert Opinion on Drug Metabolism & 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.
openaire   +3 more sources

Sapropterin dihydrochloride for phenylketonuria and tetrahydrobiopterin deficiency

Expert Review of Endocrinology & 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.
openaire   +2 more sources

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