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Items: 1 to 20 of 169

1.

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; Phenylketonuria Demographics Outcomes and Safety (PKUDOS) registry; Maternal Phenylketonuria Observational Program (PKU MOMS) sub-registry.

Mol Genet Metab. 2014 May;112(1):9-16. doi: 10.1016/j.ymgme.2014.02.016. Epub 2014 Mar 12.

2.

Use of sapropterin dihydrochloride in maternal phenylketonuria. A European experience of eight cases.

Feillet F, Muntau AC, Debray FG, Lotz-Havla AS, Puchwein-Schwepcke A, Fofou-Caillierez MB, van Spronsen F, Trefz FF.

J Inherit Metab Dis. 2014 Sep;37(5):753-62. doi: 10.1007/s10545-014-9716-5. Epub 2014 May 1.

PMID:
24789341
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. Epub 2007 Sep 12.

PMID:
17846916
4.

Potential role of tetrahydrobiopterin in the treatment of maternal phenylketonuria.

Trefz FK, Blau N.

Pediatrics. 2003 Dec;112(6 Pt 2):1566-9.

PMID:
14654666
5.

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.

6.

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
7.

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
8.

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.

9.

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
10.

A new case of maternal phenylketonuria treated with sapropterin dihydrochloride (6R-BH4).

Aldámiz-Echevarría L, Couce ML, Llarena M, Andrade F.

Gynecol Endocrinol. 2014 Oct;30(10):691-3. doi: 10.3109/09513590.2014.928688. Epub 2014 Jun 13.

PMID:
24927077
11.

[Open, non-comparative phase III clinical study to evaluate the efficacy and safety of sapropterin in patients with phenylketonuria and hyperphenylalaninemia].

Bushueva TV, Kuzenkova LM, Borovik TÉ, Nazarenko LP, Seitova GN, Filimonova MN, Pichkur NA, Samonenko NV, Shkurko TA, Akhmadeeva ÉN, Mardanova AK, Garifullina ÉR, Kovtun OP, Bazhenova IuL, Alimova IL, Kostiakova EA, Minaĭcheva LI, Saliukova OA, Sivokha VM, Rozenson OL.

Vestn Ross Akad Med Nauk. 2014;(7-8):69-77. Russian.

PMID:
25563006
12.

Role of nutrition in pregnancy with phenylketonuria and birth defects.

Matalon KM, Acosta PB, Azen C.

Pediatrics. 2003 Dec;112(6 Pt 2):1534-6.

PMID:
14654660
13.

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
14.

[The maternal phenylketonuria syndrom--still current problem].

Didycz B, Domagała L, Pietrzyk JJ.

Przegl Lek. 2009;66(1-2):4-10. Polish.

PMID:
19485248
15.

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
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; 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
17.

Long-term safety and efficacy of sapropterin: the PKUDOS registry experience.

Longo N, Arnold GL, Pridjian G, Enns GM, Ficicioglu C, Parker S, Cohen-Pfeffer JL; Phenylketonuria Demographics, Outcomes and Safety Registry.

Mol Genet Metab. 2015 Apr;114(4):557-63. doi: 10.1016/j.ymgme.2015.02.003. Epub 2015 Feb 16.

18.

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. doi: 10.1016/j.ymgme.2013.04.017. Epub 2013 May 1.

PMID:
23712020
19.

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 30;11:218. doi: 10.1186/1477-7525-11-218.

20.

Recommendations for the use of sapropterin in phenylketonuria.

Cunningham A, Bausell H, Brown M, Chapman M, DeFouw K, Ernst S, McClure J, McCune H, O'Steen D, Pender A, Skrabal J, Wessel A, Jurecki E, Shediac R, Prasad S, Gillis J, Cederbaum S.

Mol Genet Metab. 2012 Jul;106(3):269-76. doi: 10.1016/j.ymgme.2012.04.004. Epub 2012 Apr 13.

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