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Items: 1 to 20 of 97

1.

Long-term outcomes of enzyme replacement therapy for Taiwanese patients with Mucopolysaccharidosis I.

Lin HY, Lee CL, Chuang CK, Lin SP.

Pediatr Neonatol. 2019 May 29. pii: S1875-9572(19)30204-9. doi: 10.1016/j.pedneo.2019.05.005. [Epub ahead of print] No abstract available.

2.

Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series.

Lin HY, Chuang CK, Wang CH, Chien YH, Wang YM, Tsai FJ, Chou YY, Lin SJ, Pan HP, Niu DM, Hwu WL, Ke YY, Lin SP.

Mol Genet Metab Rep. 2016 Apr 18;7:63-9. doi: 10.1016/j.ymgmr.2016.04.003. eCollection 2016 Jun.

3.

Long-term effects of enzyme replacement therapy for Taiwanese patients with mucopolysaccharidosis IVA.

Lin HY, Chuang CK, Ke YY, Hsu CC, Chiu PC, Niu DM, Tsai FJ, Hwu WL, Lin JL, Lin SP.

Pediatr Neonatol. 2019 Jun;60(3):342-343. doi: 10.1016/j.pedneo.2018.08.005. Epub 2018 Aug 23. No abstract available.

4.

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
5.

Enzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type I.

Jameson E, Jones S, Wraith JE.

Cochrane Database Syst Rev. 2013 Sep 26;(9):CD009354. doi: 10.1002/14651858.CD009354.pub2. Review. Update in: Cochrane Database Syst Rev. 2013;(11):CD009354.

PMID:
24085657
6.

Immune tolerance after long-term enzyme-replacement therapy among patients who have mucopolysaccharidosis I.

Kakavanos R, Turner CT, Hopwood JJ, Kakkis ED, Brooks DA.

Lancet. 2003 May 10;361(9369):1608-13. Review.

PMID:
12747881
7.

12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment.

Gabrielli O, Clarke LA, Ficcadenti A, Santoro L, Zampini L, Volpi N, Coppa GV.

BMC Med Genet. 2016 Mar 10;17:19. doi: 10.1186/s12881-016-0284-4.

8.

Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation.

Sands MS, Vogler C, Torrey A, Levy B, Gwynn B, Grubb J, Sly WS, Birkenmeier EH.

J Clin Invest. 1997 Apr 1;99(7):1596-605.

9.

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI.

Brunelli MJ, Atallah ÁN, da Silva EM.

Cochrane Database Syst Rev. 2016 Mar 4;3:CD009806. doi: 10.1002/14651858.CD009806.pub2. Review.

PMID:
26943923
10.

Cardiac features and effects of enzyme replacement therapy in Taiwanese patients with Mucopolysaccharidosis IVA.

Lin HY, Chen MR, Lin SM, Hung CL, Niu DM, Chuang CK, Lin SP.

Orphanet J Rare Dis. 2018 Aug 29;13(1):148. doi: 10.1186/s13023-018-0883-6.

11.

Outcomes of Long-Term Treatment with Laronidase in Patients with Mucopolysaccharidosis Type I.

Laraway S, Mercer J, Jameson E, Ashworth J, Hensman P, Jones SA.

J Pediatr. 2016 Nov;178:219-226.e1. doi: 10.1016/j.jpeds.2016.08.033.

12.

Cerebral magnetic resonance findings during enzyme replacement therapy in mucopolysaccharidosis.

Matsubara Y, Miyazaki O, Kosuga M, Okuyama T, Nosaka S.

Pediatr Radiol. 2017 Nov;47(12):1659-1669. doi: 10.1007/s00247-017-3935-5. Epub 2017 Jul 21.

PMID:
28733853
13.

Clinical effectiveness of enzyme replacement therapy with galsulfase in mucopolysaccharidosis type VI treatment: Systematic review.

Gomes DF, Gallo LG, Leite BF, Silva RB, da Silva EN.

J Inherit Metab Dis. 2019 Jan;42(1):66-76. doi: 10.1002/jimd.12028. Review.

PMID:
30740728
14.

Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome.

Eisengart JB, Jarnes J, Ahmed A, Nestrasil I, Ziegler R, Delaney K, Shapiro E, Whitley C.

Mol Genet Metab Rep. 2017 Sep 27;13:64-68. doi: 10.1016/j.ymgmr.2017.07.012. eCollection 2017 Dec.

15.

Oxidative and nitrative stress and pro-inflammatory cytokines in Mucopolysaccharidosis type II patients: effect of long-term enzyme replacement therapy and relation with glycosaminoglycan accumulation.

Jacques CE, Donida B, Mescka CP, Rodrigues DG, Marchetti DP, Bitencourt FH, Burin MG, de Souza CF, Giugliani R, Vargas CR.

Biochim Biophys Acta. 2016 Sep;1862(9):1608-16. doi: 10.1016/j.bbadis.2016.05.021. Epub 2016 May 29.

16.

Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I.

Braunlin EA, Berry JM, Whitley CB.

Am J Cardiol. 2006 Aug 1;98(3):416-8. Epub 2006 Jun 12.

PMID:
16860035
17.

New insights in mucopolysaccharidosis type VI: neurological perspective.

Borlot F, Arantes PR, Quaio CR, Franco JF, Lourenço CM, Bertola DR, Kim CA.

Brain Dev. 2014 Aug;36(7):585-92. doi: 10.1016/j.braindev.2013.07.016. Epub 2013 Aug 21.

PMID:
23972383
18.

Bull's eye maculopathy and subfoveal deposition in two mucopolysaccharidosis type I patients on long-term enzyme replacement therapy.

Mack HG, Symons RCA, de Jong G.

Am J Ophthalmol Case Rep. 2017 Oct 4;9:1-6. doi: 10.1016/j.ajoc.2017.10.006. eCollection 2018 Mar.

19.

Substrate accumulation and extracellular matrix remodelling promote persistent upper airway disease in mucopolysaccharidosis patients on enzyme replacement therapy.

Pal AR, Mercer J, Jones SA, Bruce IA, Bigger BW.

PLoS One. 2018 Sep 18;13(9):e0203216. doi: 10.1371/journal.pone.0203216. eCollection 2018.

20.

Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA.

King B, Hassiotis S, Rozaklis T, Beard H, Trim PJ, Snel MF, Hopwood JJ, Hemsley KM.

J Neurochem. 2016 May;137(3):409-22. doi: 10.1111/jnc.13533. Epub 2016 Feb 24.

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