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Items: 1 to 20 of 240

1.

Sapropterin: a review of its use in the treatment of primary hyperphenylalaninaemia.

Sanford M, Keating GM.

Drugs. 2009;69(4):461-76. doi: 10.2165/00003495-200969040-00006. Review.

PMID:
19323589
2.

Spotlight on sapropterin in primary hyperphenylalaninemia.

Sanford M, Keating GM.

BioDrugs. 2009;23(3):201-2. doi: 10.2165/00063030-200923030-00007. Review.

PMID:
19627172
3.

Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study.

Levy HL, Milanowski A, Chakrapani A, Cleary M, Lee P, Trefz FK, Whitley CB, Feillet F, Feigenbaum AS, Bebchuk JD, Christ-Schmidt H, Dorenbaum A; Sapropterin Research Group.

Lancet. 2007 Aug 11;370(9586):504-10.

PMID:
17693179
4.

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. doi: 10.1016/j.ymgme.2011.03.020. Epub 2011 Mar 31.

PMID:
21646032
5.

Pharmacokinetics of sapropterin in patients with phenylketonuria.

Feillet F, Clarke L, Meli C, Lipson M, Morris AA, Harmatz P, Mould DR, Green B, Dorenbaum A, Giovannini M, Foehr E; Sapropterin Research Group.

Clin Pharmacokinet. 2008;47(12):817-25. doi: 10.2165/0003088-200847120-00006.

PMID:
19026037
6.

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; Sapropterin Research Group.

Am J Med Genet A. 2008 Nov 15;146A(22):2851-9. doi: 10.1002/ajmg.a.32562.

PMID:
18932221
7.

Sapropterin dihydrochloride for phenylketonuria.

Somaraju UR, Merrin M.

Cochrane Database Syst Rev. 2012 Dec 12;12:CD008005. doi: 10.1002/14651858.CD008005.pub3. Review. Update in: Cochrane Database Syst Rev. 2015;3:CD008005.

PMID:
23235653
8.

Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias.

Blau N.

Expert Opin Drug Metab Toxicol. 2013 Sep;9(9):1207-18. doi: 10.1517/17425255.2013.804064. Epub 2013 May 27. Review.

PMID:
23705856
9.

Sapropterin dihydrochloride for phenylketonuria.

Somaraju UR, Merrin M.

Cochrane Database Syst Rev. 2015 Mar 27;(3):CD008005. doi: 10.1002/14651858.CD008005.pub4. Review.

PMID:
25812600
10.

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; Sapropterin Study Group.

J Pediatr. 2009 May;154(5):700-7. doi: 10.1016/j.jpeds.2008.11.040. Epub 2009 Mar 4.

PMID:
19261295
11.

Sapropterin dihydrochloride for phenylketonuria.

Somaraju UR, Merrin M.

Cochrane Database Syst Rev. 2010 Jun 16;(6):CD008005. doi: 10.1002/14651858.CD008005.pub2. Review. Update in: Cochrane Database Syst Rev. 2012;12:CD008005.

PMID:
20556789
12.

Sapropterin: a new therapeutic agent for phenylketonuria.

Hegge KA, Horning KK, Peitz GJ, Hegge K.

Ann Pharmacother. 2009 Sep;43(9):1466-73. doi: 10.1345/aph.1M050. Epub 2009 Aug 4. Review.

PMID:
19654333
13.

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. doi: 10.1016/j.ymgme.2010.03.022. Epub 2010 Apr 3.

14.

Optimizing the use of sapropterin (BH(4)) in the management of phenylketonuria.

Blau N, Bélanger-Quintana A, Demirkol M, Feillet F, Giovannini M, MacDonald A, Trefz FK, van Spronsen FJ.

Mol Genet Metab. 2009 Apr;96(4):158-63. doi: 10.1016/j.ymgme.2009.01.002. Epub 2009 Feb 8. Review.

PMID:
19208488
16.

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. doi: 10.1542/peds.2012-3291. Epub 2013 May 20.

17.

A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria.

Burton B, Grant M, Feigenbaum A, Singh R, Hendren R, Siriwardena K, Phillips J 3rd, Sanchez-Valle A, Waisbren S, Gillis J, Prasad S, Merilainen M, Lang W, Zhang C, Yu S, Stahl S.

Mol Genet Metab. 2015 Mar;114(3):415-24. doi: 10.1016/j.ymgme.2014.11.011. Epub 2014 Nov 26.

18.

Testing for tetrahydrobiopterin responsiveness in patients with hyperphenylalaninemia due to phenylalanine hydroxylase deficiency.

Cerone R, Andria G, Giovannini M, Leuzzi V, Riva E, Burlina A.

Adv Ther. 2013 Mar;30(3):212-28. doi: 10.1007/s12325-013-0011-x. Epub 2013 Feb 20. Review.

PMID:
23436109
19.

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. Epub 2007 Sep 12.

PMID:
17846916
20.

START, a double blind, placebo-controlled pharmacogenetic test of responsiveness to sapropterin dihydrochloride in phenylketonuria patients.

Utz JR, Lorentz CP, Markowitz D, Rudser KD, Diethelm-Okita B, Erickson D, Whitley CB.

Mol Genet Metab. 2012 Feb;105(2):193-7. doi: 10.1016/j.ymgme.2011.10.014. Epub 2011 Oct 29.

PMID:
22112818

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