Results 161 to 170 of about 2,156 (206)
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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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Sapropterin: A New Therapeutic Agent for Phenylketonuria
Annals of Pharmacotherapy, 2009Objective: 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:
Karly A, Hegge +3 more
openaire +2 more sources
A Systematic Review of BH4 (Sapropterin) for the Adjuvant Treatment of Phenylketonuria [PDF]
JIMD Reports, 2012 Dietary management is the mainstay of effective treatment in PKU, but dietary restriction is difficult and additional treatment options are needed.To systematically review evidence regarding sapropterin (BH4) use as an adjunct to dietary restriction in individuals with PKU.Five databases including MEDLINE up to August 2011.Two reviewers independently ...
Christopher J Fonnesbeck +2 more
exaly +3 more sources
The effects of sapropterin on urinary monoamine metabolites in phenylketonuria
Molecular Genetics and Metabolism, 2013Sapropterin dihydrochloride (BH4, tetrahydrobiopterin) can lower plasma phenylalanine (Phe) concentrations for a subset of patients with phenylketonuria (PKU), an inborn error of metabolism. Studies suggest that monoamine neurotransmitter concentrations are low in PKU patients.
Teresa D, Douglas +3 more
openaire +2 more sources
Use of sapropterin dihydrochloride in maternal phenylketonuria. A European experience of eight cases
Journal of Inherited Metabolic Disease, 2014AbstractSapropterin dihydrochloride (SD) is the first drug treatment for phenylketonuria (PKU), but due to the lack of data, its use in maternal PKU must be undertaken with caution as noted in the FDA and EMEA labels. We collected data from eight pregnancies in PKU women treated with SD and we analysed the phenotypes of these patients, their ...
Feillet, François +7 more
openaire +3 more sources
Journal of Pediatric Endocrinology & Metabolism (JPEM), 2019
Background Phenylketonuria (PKU) is one of the most common types of inborn error of metabolism. The mainstay of therapy for PKU has been dietary phenylalanine (Phe) restriction.
P. Eshraghi +3 more
semanticscholar +1 more source
Background Phenylketonuria (PKU) is one of the most common types of inborn error of metabolism. The mainstay of therapy for PKU has been dietary phenylalanine (Phe) restriction.
P. Eshraghi +3 more
semanticscholar +1 more source
Sapropterin. Phenylketonuria: for a minority of patients.
Prescrire international, 2010Phenylketonuria and tetrahydrobiopterin deficiency are rare congenital disorders involving phenylalanine metabolism. If left untreated, they lead to severe mental retardation and neuropsychological problems. Management is based on a diet low in phenylalanine, started as early as possible.
openaire +1 more source
Pegvaliase therapy in sapropterin-responsive PKU patients
Molecular Genetics and Metabolism, 2022E. Vucko +7 more
semanticscholar +1 more source