Results 11 to 20 of about 2,156 (206)

Characterization of Sapropterin Dihydrochloride Tablet [PDF]

Journal of Drug Delivery and Therapeutics
Characterization 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
semanticscholar   +3 more sources

Sapropterin (BH4) Aggravates Autoimmune Encephalomyelitis in Mice [PDF]

Neurotherapeutics, 2021
Depletion of the enzyme cofactor, tetrahydrobiopterin (BH4), in T-cells was shown to prevent their proliferation upon receptor stimulation in models of allergic inflammation in mice, suggesting that BH4 drives autoimmunity. Hence, the clinically available BH4 drug (sapropterin) might increase the risk of autoimmune diseases.
Katja Schmitz, Sandra Trautmann, Lisa Hahnefeld, Caroline Fischer, Yannick Schreiber, Annett Wilken-Schmitz, Robert Gurke, Robert Brunkhorst, Ernst R. Werner, Katrin Watschinger, Sabine Wicker, Dominique Thomas, Gerd Geisslinger, Irmgard Tegeder   +13 more
semanticscholar   +5 more sources

Sapropterin hydrochloride: enzyme enhancement therapy for phenylketonuria [PDF]

Therapeutic Advances in Endocrinology and Metabolism, 2011
Phenylketonuria (PKU) is an inherited disorder of amino acid metabolism caused by deficiency of the enzyme phenylalanine hydroxylase (PAH). Historically PKU was a common genetic cause of severe learning difficulties and developmental delay, but with the ...
Robin Lachmann
doaj   +5 more sources

Use of sapropterin in Mexican patients with yperphenylalaninemia

Acta Pediátrica de México, 2014
Hyperphenylalaninemia is caused by deficient enzyme activity of phenylalanine hydroxylase. It was one of the first genetic disorders susceptible to treatment with a natural protein restricted diet for life.
Susana Monroy-Santoyo, Leticia Belmont-Martínez, Cynthia Fernández-Lainez   +2 more
doaj   +3 more sources

HPLC-Based Analysis of Impurities in Sapropterin Branded and Generic Tablets

Pharmaceutics, 2020
This work was aimed at the definition of a chromatographic method able to separate and quantify impurities present in sapropterin-containing drugs during an accelerated stability study. The chromatographic method was applied to the orphan drug Kuvan® and
Emanuela Scudellaro, Luciana Tartaglione, Fabio Varriale, Carmela Dell’Aversano, Orazio Taglialatela-Scafati   +4 more
doaj   +4 more sources

Improving sapropterin administration efficacy in PKU: Clinical practice case studies. [PDF]

Mol Genet Metab Rep
In patients with phenylketonuria (PKU), sapropterin dihydrochloride (sapropterin) lowers blood phenylalanine (Phe) and may enhance Phe tolerance in sapropterin responsive patients.
Tosi M, Daly A, Ashmore C, Pinto A, Vijay S, Verduci E, Evans S, MacDonald A.   +7 more
europepmc   +3 more sources

Protein and Micronutrient Intake After Two Years of Sapropterin Treatment in PKU. [PDF]

Nutrients
Background: Sapropterin allows dietary liberalisation in responsive individuals with phenylketonuria (PKU), increasing natural protein intake and reducing dependence on protein substitutes (PSs).
Yilmaz Nas O, Ashmore C, Gama MI, Daly A, Evans S, Pinto A, Ozdogan Y, MacDonald A.   +7 more
europepmc   +3 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, 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
openaire   +4 more sources

Sapropterin Dihydrochloride Responsiveness in Phenylketonuria: A Case Series Exploring Gaps in Comprehensive Patient Monitoring. [PDF]

Nutrients
Background: Phenylketonuria (PKU) is a rare autosomal recessive metabolic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene, leading to hyperphenylalaninemia (HPA).
Lo Bianco M, Leonardi R, Migliore A, Moliteo E, Sciacca M, Rinella S, Pappalardo MG, La Spina L, Messina M, Iacobacci R, Ruggieri M, Meli C, Polizzi A.   +12 more
europepmc   +3 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.
Blau, Nenad
openaire   +4 more sources