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Items: 1 to 20 of 94

1.

Ten years of enzyme replacement therapy in paediatric onset mucopolysaccharidosis II in England.

Broomfield A, Davison J, Roberts J, Stewart C, Hensman P, Beesley C, Tylee K, Rust S, Schwahn B, Jameson E, Vijay S, Santra S, Sreekantam S, Ramaswami U, Chakrapani A, Raiman J, Cleary MA, Jones SA.

Mol Genet Metab. 2019 Jul 30. pii: S1096-7192(19)30459-7. doi: 10.1016/j.ymgme.2019.07.016. [Epub ahead of print]

PMID:
31383595
2.

First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: case observations from the Hunter Outcome Survey (HOS).

Alcalde-Martín C, Muro-Tudelilla JM, Cancho-Candela R, Gutiérrez-Solana LG, Pintos-Morell G, Martí-Herrero M, Munguira-Aguado P, Galán-Gómez E.

Eur J Med Genet. 2010 Nov-Dec;53(6):371-7. doi: 10.1016/j.ejmg.2010.07.013. Epub 2010 Aug 10.

PMID:
20709629
3.

Clinical efficacy of enzyme replacement therapy in paediatric Hunter patients, an independent study of 3.5 years.

Tomanin R, Zanetti A, D'Avanzo F, Rampazzo A, Gasparotto N, Parini R, Pascarella A, Concolino D, Procopio E, Fiumara A, Borgo A, Frigo AC, Scarpa M.

Orphanet J Rare Dis. 2014 Sep 18;9:129. doi: 10.1186/s13023-014-0129-1.

4.

The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.

Wyatt K, Henley W, Anderson L, Anderson R, Nikolaou V, Stein K, Klinger L, Hughes D, Waldek S, Lachmann R, Mehta A, Vellodi A, Logan S.

Health Technol Assess. 2012;16(39):1-543. doi: 10.3310/hta16390.

5.
6.

Efficacy of Idursulfase therapy in patients with Mucopolysaccharidosis type II who initiated enzyme replacement therapy in adult age. A systematic review of the literature.

Pérez-López J, Moltó-Abad M, Muñoz-Delgado C, Morales-Conejo M, Ceberio-Hualde L, Del Toro M.

Mol Genet Metab. 2018 Jul;124(3):216-227. doi: 10.1016/j.ymgme.2018.04.013. Epub 2018 Apr 30.

PMID:
29801985
7.

Activities of daily living in patients with Hunter syndrome: impact of enzyme replacement therapy and hematopoietic stem cell transplantation.

Tanjuakio J, Suzuki Y, Patel P, Yasuda E, Kubaski F, Tanaka A, Yabe H, Mason RW, Montaño AM, Orii KE, Orii KO, Fukao T, Orii T, Tomatsu S.

Mol Genet Metab. 2015 Feb;114(2):161-9. doi: 10.1016/j.ymgme.2014.11.002. Epub 2014 Nov 8.

8.

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
9.

Enzymatic replacement therapy for Hunter disease: Up to 9 years experience with 17 patients.

Parini R, Rigoldi M, Tedesco L, Boffi L, Brambilla A, Bertoletti S, Boncimino A, Del Longo A, De Lorenzo P, Gaini R, Gallone D, Gasperini S, Giussani C, Grimaldi M, Grioni D, Meregalli P, Messinesi G, Nichelli F, Romagnoli M, Russo P, Sganzerla E, Valsecchi G, Biondi A.

Mol Genet Metab Rep. 2015 Apr 22;3:65-74. doi: 10.1016/j.ymgmr.2015.03.011. eCollection 2015 Jun.

10.

Effects of idursulfase enzyme replacement therapy for Mucopolysaccharidosis type II when started in early infancy: comparison in two siblings.

Tajima G, Sakura N, Kosuga M, Okuyama T, Kobayashi M.

Mol Genet Metab. 2013 Mar;108(3):172-7. doi: 10.1016/j.ymgme.2012.12.010. Epub 2013 Jan 9.

PMID:
23375472
11.

Impact of enzyme replacement therapy on linear growth in Korean patients with mucopolysaccharidosis type II (Hunter syndrome).

Cho SY, Huh R, Chang MS, Lee J, Kwun Y, Maeng SH, Kim SJ, Sohn YB, Park SW, Kwon EK, Han SJ, Jung J, Jin DK.

J Korean Med Sci. 2014 Feb;29(2):254-60. doi: 10.3346/jkms.2014.29.2.254. Epub 2014 Jan 28.

12.

Clinical response to long term enzyme replacement treatment in children, adolescent and adult patients with Hunter syndrome.

Dalmau Serra J, Vitoria Miñana I, Calderón Fernández R, Cortell Aznar I.

Med Clin (Barc). 2015 Nov 6;145(9):392-8. doi: 10.1016/j.medcli.2015.06.015. Epub 2015 Sep 8.

PMID:
26360015
13.

Cardiac structure and function and effects of enzyme replacement therapy in patients with mucopolysaccharidoses I, II, IVA and VI.

Lin HY, Chuang CK, Chen MR, Lin SM, Hung CL, Chang CY, Chiu PC, Tsai WH, Niu DM, Tsai FJ, Lin SJ, Hwu WL, Lin JL, Lin SP.

Mol Genet Metab. 2016 Apr;117(4):431-7. doi: 10.1016/j.ymgme.2016.02.003. Epub 2016 Feb 16.

PMID:
26899310
14.

Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS).

Muenzer J, Giugliani R, Scarpa M, Tylki-Szymańska A, Jego V, Beck M.

Orphanet J Rare Dis. 2017 Oct 3;12(1):161. doi: 10.1186/s13023-017-0712-3.

15.

Impact of Enzyme Replacement Therapy and Hematopoietic Stem Cell Therapy on Growth in Patients with Hunter Syndrome.

Patel P, Suzuki Y, Tanaka A, Yabe H, Kato S, Shimada T, Mason RW, Orii KE, Fukao T, Orii T, Tomatsu S.

Mol Genet Metab Rep. 2014;1:184-196.

16.

Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study.

Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P.

Am J Med Genet A. 2014 Aug;164A(8):1953-64. doi: 10.1002/ajmg.a.36584. Epub 2014 Apr 24.

17.

Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II.

Schulze-Frenking G, Jones SA, Roberts J, Beck M, Wraith JE.

J Inherit Metab Dis. 2011 Feb;34(1):203-8. doi: 10.1007/s10545-010-9215-2. Epub 2010 Oct 27.

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. 2016 Feb 5;2:CD008185. doi: 10.1002/14651858.CD008185.pub4. Review.

PMID:
26845288
19.

Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.

Tomatsu S, Sawamoto K, Alméciga-Díaz CJ, Shimada T, Bober MB, Chinen Y, Yabe H, Montaño AM, Giugliani R, Kubaski F, Yasuda E, Rodríguez-López A, Espejo-Mojica AJ, Sánchez OF, Mason RW, Barrera LA, Mackenzie WG, Orii T.

Drug Des Devel Ther. 2015 Apr 1;9:1937-53. doi: 10.2147/DDDT.S68562. eCollection 2015. Review.

20.

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. Update in: Cochrane Database Syst Rev. 2016;2:CD008185.

PMID:
24399699

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