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Results: 1 to 20 of 85

1.

[Efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II with and without comparison to placebo: systematic review and meta-analysis].

Alegra T, Eizerik DP, de Cerqueira CC, Pereira TV, Dornelles AD, Schwartz IV.

Cad Saude Publica. 2013 Nov;29 Suppl 1:S45-58. Review. Portuguese.

2.

Mucopolysaccharidosis type II, Hunter's syndrome.

Tylki-Szymańska A.

Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:107-13. Review.

PMID:
25345092
3.

[Shift of focus in the financing of Hungarian drugs. Reimbursement for orphan drugs for treating rare diseases: financing of enzyme replacement therapy in Hungary].

Szegedi M, Molnár MJ, Boncz I, Kosztolányi G.

Orv Hetil. 2014 Nov 2;155(44):1735-41. doi: 10.1556/OH.2014.30031. Review. Hungarian.

PMID:
25344850
4.

Current and potential therapeutic strategies for mucopolysaccharidoses.

Noh H, Lee JI.

J Clin Pharm Ther. 2014 Jun;39(3):215-24. doi: 10.1111/jcpt.12136. Epub 2014 Feb 25. Review.

PMID:
24612142
5.

Treatment of lysosomal storage diseases: recent patents and future strategies.

Ortolano S, Viéitez I, Navarro C, Spuch C.

Recent Pat Endocr Metab Immune Drug Discov. 2014 Jan;8(1):9-25. Review.

PMID:
24433521
6.

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome).

da Silva EM, Strufaldi MW, Andriolo RB, Silva LA.

Cochrane Database Syst Rev. 2014 Jan 8;1:CD008185. doi: 10.1002/14651858.CD008185.pub3. Review.

PMID:
24399699
7.

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses.

Muenzer J.

Mol Genet Metab. 2014 Feb;111(2):63-72. doi: 10.1016/j.ymgme.2013.11.015. Epub 2013 Dec 11. Review.

8.

Neurologic manifestations of inherited disorders of connective tissue.

Debette S, Germain DP.

Handb Clin Neurol. 2014;119:565-76. doi: 10.1016/B978-0-7020-4086-3.00037-0. Review.

PMID:
24365320
9.

Radiological and clinical characterization of the lysosomal storage disorders: non-lipid disorders.

Parker EI, Xing M, Moreno-De-Luca A, Harmouche E, Terk MR.

Br J Radiol. 2014 Jan;87(1033):20130467. doi: 10.1259/bjr.20130467. Epub 2013 Nov 14. Review. Erratum in: Br J Radiol. 2014 Mar;87(1035):20149002.

10.

Boning up on autophagy: the role of autophagy in skeletal biology.

Shapiro IM, Layfield R, Lotz M, Settembre C, Whitehouse C.

Autophagy. 2014 Jan;10(1):7-19. doi: 10.4161/auto.26679. Epub 2013 Nov 11. Review.

11.

[Clinical practice guideline for the management of Hunter syndrome. Hunter España working group].

Guillén-Navarro E, Blasco AJ, Gutierrez-Solana LG, Couce ML, Cancho-Candela R, Lázaro P; grupo de trabajo Hunter España.

Med Clin (Barc). 2013 Nov 16;141(10):453.e1-13. doi: 10.1016/j.medcli.2013.07.010. Epub 2013 Sep 21. Spanish. No abstract available.

PMID:
24060500
12.

Osteoimmunology in mucopolysaccharidoses type I, II, VI and VII. Immunological regulation of the osteoarticular system in the course of metabolic inflammation.

Opoka-Winiarska V, Jurecka A, Emeryk A, Tylki-Szymańska A.

Osteoarthritis Cartilage. 2013 Dec;21(12):1813-23. doi: 10.1016/j.joca.2013.08.001. Epub 2013 Aug 13. Review.

13.

Enzyme replacement therapy for lysosomal storage diseases.

Ohashi T.

Pediatr Endocrinol Rev. 2012 Oct;10 Suppl 1:26-34. Review.

PMID:
23330243
14.

Human pathology in NCL.

Anderson GW, Goebel HH, Simonati A.

Biochim Biophys Acta. 2013 Nov;1832(11):1807-26. doi: 10.1016/j.bbadis.2012.11.014. Epub 2012 Nov 29. Review.

15.

Sulfatase inhibitors: a patent review.

Williams SJ.

Expert Opin Ther Pat. 2013 Jan;23(1):79-98. doi: 10.1517/13543776.2013.736965. Epub 2012 Nov 8. Review.

PMID:
23136854
16.

Effect of rapid cessation of enzyme replacement therapy: a report of 5 cases and a review of the literature.

Jurecka A, Żuberuber Z, Opoka-Winiarska V, Węgrzyn G, Tylki-Szymańska A.

Mol Genet Metab. 2012 Nov;107(3):508-12. doi: 10.1016/j.ymgme.2012.08.013. Epub 2012 Aug 17. Review.

PMID:
22926198
17.

Therapy for the mucopolysaccharidoses.

Valayannopoulos V, Wijburg FA.

Rheumatology (Oxford). 2011 Dec;50 Suppl 5:v49-59. doi: 10.1093/rheumatology/ker396. Review.

18.

Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome).

da Silva EM, Strufaldi MW, Andriolo RB, Silva LA.

Cochrane Database Syst Rev. 2011 Nov 9;(11):CD008185. doi: 10.1002/14651858.CD008185.pub2. Review. Update in: Cochrane Database Syst Rev. 2014;1:CD008185.

PMID:
22071845
19.

The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus.

Muenzer J, Bodamer O, Burton B, Clarke L, Frenking GS, Giugliani R, Jones S, Rojas MV, Scarpa M, Beck M, Harmatz P.

Eur J Pediatr. 2012 Jan;171(1):181-8. doi: 10.1007/s00431-011-1606-3. Epub 2011 Oct 29.

20.

[Lysosomal storage diseases--an overview].

Kloska A, Tylki-Szymańska A, Wegrzyn G.

Postepy Biochem. 2011;57(2):128-32. Review. Polish.

PMID:
21913413
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