Results 181 to 190 of about 2,204 (208)

Patient voices on PKU care: Insights from focus groups with current and former patients. [PDF]

Mol Genet Metab Rep
Zhu E, Hollander S, Sacharow S.
europepmc   +1 more source

Specific drugs for rare diseases in a province of eastern China under catalog management: from 2021 to 2023. [PDF]

Front Pharmacol
Nie R, Zhao Z, Zhang Y, Xu B, Zhang W.
europepmc   +1 more source

Assessing sleep habits in school-aged children with phenylketonuria: a comparative study. [PDF]

Eur J Pediatr
Mete Yeşil A, Akar HT, Yıldız Y, Sivri S, Özmert EN.   +4 more
europepmc   +1 more source

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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, L. Clarke, C. Meli, M. Lipson, A. A. Morris, P. Harmatz, D. R. Mould, B. Green, A. Dorenbaum, M. Giovannini, E. Foehr, Sapropterin Research Group   +11 more
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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 ...
Gillian M. Keating, Mark Sanford
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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/
Danial E. Baker, Dennis J. Cada, Terri Levien   +2 more
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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, Kristin K Horning, Gregory Peitz, Kassy Hegge   +3 more
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Optimizing the use of sapropterin (BH4) in the management of phenylketonuria

Molecular Genetics and Metabolism, 2009
Phenylketonuria (PKU) is caused by mutations in the phenylalanine hydroxylase (PAH) gene, leading to deficient conversion of phenylalanine (Phe) to tyrosine and accumulation of toxic levels of Phe. A Phe-restricted diet is essential to reduce blood Phe levels and prevent long-term neurological impairment and other adverse sequelae.
Blau, N, Bélanger-Quintana, A, Demirkol, M, Feillet, F, Giovannini, M, MacDonald, A, Trefz, F K, van Spronsen, F J   +7 more
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