BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

450 related articles for article (PubMed ID: 20418136)

  • 1. Introduction of sapropterin dihydrochloride as standard of care in patients with phenylketonuria.
    Vernon HJ; Koerner CB; Johnson MR; Bergner A; Hamosh A
    Mol Genet Metab; 2010 Jul; 100(3):229-33. PubMed ID: 20418136
    [TBL] [Abstract][Full Text] [Related]  

  • 2. The effects of sapropterin on urinary monoamine metabolites in phenylketonuria.
    Douglas TD; Jinnah HA; Bernhard D; Singh RH
    Mol Genet Metab; 2013 Jul; 109(3):243-50. PubMed ID: 23712020
    [TBL] [Abstract][Full Text] [Related]  

  • 3. The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R-tetrahydrobiopterin): a phase II, multicentre, open-label, screening study.
    Burton BK; Grange DK; Milanowski A; Vockley G; Feillet F; Crombez EA; Abadie V; Harding CO; Cederbaum S; Dobbelaere D; Smith A; Dorenbaum A
    J Inherit Metab Dis; 2007 Oct; 30(5):700-7. PubMed ID: 17846916
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Long-term comparative effectiveness of pegvaliase versus standard of care comparators in adults with phenylketonuria.
    Zori R; Ahring K; Burton B; Pastores GM; Rutsch F; Jha A; Jurecki E; Rowell R; Harding C
    Mol Genet Metab; 2019; 128(1-2):92-101. PubMed ID: 31439512
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial.
    Muntau AC; Burlina A; Eyskens F; Freisinger P; De Laet C; Leuzzi V; Rutsch F; Sivri HS; Vijay S; Bal MO; Gramer G; Pazdírková R; Cleary M; Lotz-Havla AS; Munafo A; Mould DR; Moreau-Stucker F; Rogoff D
    Orphanet J Rare Dis; 2017 Mar; 12(1):47. PubMed ID: 28274234
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride.
    Douglas TD; Ramakrishnan U; Kable JA; Singh RH
    Health Qual Life Outcomes; 2013 Dec; 11():218. PubMed ID: 24373161
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Individualized long-term outcomes in blood phenylalanine concentrations and dietary phenylalanine tolerance in 11 patients with primary phenylalanine hydroxylase (PAH) deficiency treated with Sapropterin-dihydrochloride.
    Stockler-Ipsiroglu S; Yuskiv N; Salvarinova R; Apatean D; Ho G; Cheng B; Giezen A; Lillquist Y; Ueda K
    Mol Genet Metab; 2015 Mar; 114(3):409-14. PubMed ID: 25497838
    [TBL] [Abstract][Full Text] [Related]  

  • 8. PTC923 (sepiapterin) lowers elevated blood phenylalanine in subjects with phenylketonuria: a phase 2 randomized, multi-center, three-period crossover, open-label, active controlled, all-comers study.
    Bratkovic D; Margvelashvili L; Tchan MC; Nisbet J; Smith N
    Metabolism; 2022 Mar; 128():155116. PubMed ID: 34973284
    [TBL] [Abstract][Full Text] [Related]  

  • 9. International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria.
    Muntau AC; Adams DJ; Bélanger-Quintana A; Bushueva TV; Cerone R; Chien YH; Chiesa A; Coşkun T; de Las Heras J; Feillet F; Katz R; Lagler F; Piazzon F; Rohr F; van Spronsen FJ; Vargas P; Wilcox G; Bhattacharya K
    Mol Genet Metab; 2019 May; 127(1):1-11. PubMed ID: 31103398
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria.
    Lee P; Treacy EP; Crombez E; Wasserstein M; Waber L; Wolff J; Wendel U; Dorenbaum A; Bebchuk J; Christ-Schmidt H; Seashore M; Giovannini M; Burton BK; Morris AA;
    Am J Med Genet A; 2008 Nov; 146A(22):2851-9. PubMed ID: 18932221
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Sapropterin: a review of its use in the treatment of primary hyperphenylalaninaemia.
    Sanford M; Keating GM
    Drugs; 2009; 69(4):461-76. PubMed ID: 19323589
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial.
    Muntau AC; Burlina A; Eyskens F; Freisinger P; Leuzzi V; Sivri HS; Gramer G; Pazdírková R; Cleary M; Lotz-Havla AS; Lane P; Alvarez I; Rutsch F
    Orphanet J Rare Dis; 2021 Aug; 16(1):341. PubMed ID: 34344399
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Safety of extended treatment with sapropterin dihydrochloride in patients with phenylketonuria: results of a phase 3b study.
    Burton BK; Nowacka M; Hennermann JB; Lipson M; Grange DK; Chakrapani A; Trefz F; Dorenbaum A; Imperiale M; Kim SS; Fernhoff PM
    Mol Genet Metab; 2011 Aug; 103(4):315-22. PubMed ID: 21646032
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Long-term follow-up and outcome of phenylketonuria patients on sapropterin: a retrospective study.
    Keil S; Anjema K; van Spronsen FJ; Lambruschini N; Burlina A; Bélanger-Quintana A; Couce ML; Feillet F; Cerone R; Lotz-Havla AS; Muntau AC; Bosch AM; Meli CA; Billette de Villemeur T; Kern I; Riva E; Giovannini M; Damaj L; Leuzzi V; Blau N
    Pediatrics; 2013 Jun; 131(6):e1881-8. PubMed ID: 23690520
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias.
    Blau N
    Expert Opin Drug Metab Toxicol; 2013 Sep; 9(9):1207-18. PubMed ID: 23705856
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study.
    Trefz FK; Burton BK; Longo N; Casanova MM; Gruskin DJ; Dorenbaum A; Kakkis ED; Crombez EA; Grange DK; Harmatz P; Lipson MH; Milanowski A; Randolph LM; Vockley J; Whitley CB; Wolff JA; Bebchuk J; Christ-Schmidt H; Hennermann JB;
    J Pediatr; 2009 May; 154(5):700-7. PubMed ID: 19261295
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Use of sapropterin in the management of phenylketonuria: seven case reports.
    Gokmen Ozel H; Lammardo AM; Motzfeldt K; Robert M; Rocha JC; van Rijn M; Ahring K; Bélanger-Quintana A; MacDonald A; Dokoupil K
    Mol Genet Metab; 2013 Feb; 108(2):109-11. PubMed ID: 23266371
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Response to sapropterin hydrochloride (Kuvan®) in children with phenylketonuria (PKU): a clinical trial.
    Eshraghi P; Noroozi Asl S; Bagheri S; Chalak V
    J Pediatr Endocrinol Metab; 2019 Aug; 32(8):885-888. PubMed ID: 31237861
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Sapropterin dihydrochloride for phenylketonuria.
    Somaraju UR; Merrin M
    Cochrane Database Syst Rev; 2015 Mar; 2015(3):CD008005. PubMed ID: 25812600
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Sapropterin dihydrochloride use in pregnant women with phenylketonuria: an interim report of the PKU MOMS sub-registry.
    Grange DK; Hillman RE; Burton BK; Yano S; Vockley J; Fong CT; Hunt J; Mahoney JJ; Cohen-Pfeffer JL; ;
    Mol Genet Metab; 2014 May; 112(1):9-16. PubMed ID: 24667082
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 23.