-
1
-
-
84969536912
-
Long-term weekly dosing of idursulfase in the treatment of mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]
-
Beck M, Wraith E, Muenzer J, Giugliani R, Harmatz P, Eng CM, et al. Long-term weekly dosing of idursulfase in the treatment of mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2007;30(Suppl 1):116.
-
(2007)
Journal of Inherited Metabolic Disease
, vol.30
, pp. 116
-
-
Beck, M.1
Wraith, E.2
Muenzer, J.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
-
2
-
-
79951578486
-
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
-
Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, et al. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genetics in Medicine 2011;13(2):95-101.
-
(2011)
Genetics in Medicine
, vol.13
, Issue.2
, pp. 95-101
-
-
Muenzer, J.1
Beck, M.2
Eng, C.M.3
Giugliani, R.4
Harmatz, P.5
Martin, R.6
-
3
-
-
84969528514
-
Clinical benefit of enzyme replacement therapy (ERT) in mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]
-
Muenzer J, Wraith E, Beck M, Giugliani R, Harmatz P, Eng CM, et al. Clinical benefit of enzyme replacement therapy (ERT) in mucopolysaccharidosis II (MPS II, Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2006;29(Suppl 1):28.
-
(2006)
Journal of Inherited Metabolic Disease
, vol.29
, pp. 28
-
-
Muenzer, J.1
Wraith, E.2
Beck, M.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
-
4
-
-
33747209013
-
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
-
Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genetics in Medicine 2006;8(8):465-73.
-
(2006)
Genetics in Medicine
, vol.8
, Issue.8
, pp. 465-473
-
-
Muenzer, J.1
Wraith, J.E.2
Beck, M.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
-
5
-
-
37249033169
-
Clinical study of enzyme replacement therapy with idursulfase
-
Gutiérrez-Solana LG. Clinical study of enzyme replacement therapy with idursulfase. Revista de Neurologia 2007;44(Suppl 1):S7-S11.
-
(2007)
Revista de Neurologia
, vol.44
, pp. S7-S11
-
-
Gutiérrez-Solana, L.G.1
-
7
-
-
33846899175
-
A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)
-
Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Molecular Genetics and Metabolism. 2007;90(3):329-37.
-
(2007)
Molecular Genetics and Metabolism.
, vol.90
, Issue.3
, pp. 329-337
-
-
Muenzer, J.1
Gucsavas-Calikoglu, M.2
McCandless, S.E.3
Schuetz, T.J.4
Kimura, A.5
-
8
-
-
84969555145
-
The 12-month experience of enzyme replacement for mucopolysaccharidosis II (Hunter syndrome) [abstract]
-
Muenzer J, Towle D, Calikoglu M, McCandless S. The 12-month experience of enzyme replacement for mucopolysaccharidosis II (Hunter syndrome) [abstract]. Journal of Inherited Metabolic Disease 2003;26(Suppl 2):144.
-
(2003)
Journal of Inherited Metabolic Disease
, vol.26
, pp. 144
-
-
Muenzer, J.1
Towle, D.2
Calikoglu, M.3
McCandless, S.4
-
9
-
-
84875017970
-
Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome)
-
Sohn YB, Cho SY, Park SW, Kim SJ, Ko AR, Kwon EK, et al. Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome). Orphanet Journal of Rare Diseases 2013;8:42. [DOI: ]
-
(2013)
Orphanet Journal of Rare Diseases
, vol.8
, pp. 42
-
-
Sohn, Y.B.1
Cho, S.Y.2
Park, S.W.3
Kim, S.J.4
Ko, A.R.5
Kwon, E.K.6
-
10
-
-
84969520001
-
A clinical trial of idursulfase in Hunter syndrome patients 5 years old and younger [abstract]
-
Tylki-Szymanska A, Giugliani R, Hwu WL. A clinical trial of idursulfase in Hunter syndrome patients 5 years old and younger [abstract]. Journal of Inherited Metabolic Disease 2008;31(Suppl 1):130.
-
(2008)
Journal of Inherited Metabolic Disease
, vol.31
, pp. 130
-
-
Tylki-Szymanska, A.1
Giugliani, R.2
Hwu, W.L.3
-
11
-
-
78349313662
-
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, et al. 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). European Journal of Medical Genetics 2010;53(6):371-7.
-
(2010)
European Journal of Medical Genetics
, vol.53
, Issue.6
, pp. 371-377
-
-
Alcalde-Martín, C.1
Muro-Tudelilla, J.M.2
Cancho-Candela, R.3
Gutiérrez-Solana, L.G.4
Pintos-Morell, G.5
Martí-Herrero, M.6
-
12
-
-
0036644465
-
ATS statement: guidelines for the six-minute walk test
-
American Thoracic Society.
-
American Thoracic Society. ATS statement: guidelines for the six-minute walk test. American Journal of Respiratory and Critical Care Medicine 2002;166(1):111-7.
-
(2002)
American Journal of Respiratory and Critical Care Medicine
, vol.166
, Issue.1
, pp. 111-117
-
-
-
13
-
-
0026872139
-
Measuring urinary glycosaminoglycans in the presence of protein: an improved screening procedure for mucopolysaccharidoses based on dimethylmethylene blue
-
de Jong JG, Wevers RA, Liebrand-van Sambeek R. Measuring urinary glycosaminoglycans in the presence of protein: an improved screening procedure for mucopolysaccharidoses based on dimethylmethylene blue. Clinical Chemistry 1992;38(6):803-7.
-
(1992)
Clinical Chemistry
, vol.38
, Issue.6
, pp. 803-807
-
-
de Jong, J.G.1
Wevers, R.A.2
Liebrand-van Sambeek, R.3
-
14
-
-
0036211922
-
Meta-analyses involving cross-over trials: methodological issues
-
[PUBMED: 11914310]
-
Elbourne DR, Altman DG, Higgins JP, Curtin F, Worthington HV, Vail A. Meta-analyses involving cross-over trials: methodological issues. International Journal of Epidemiology 2002;31(1):140-9. [PUBMED: 11914310]
-
(2002)
International Journal of Epidemiology
, vol.31
, Issue.1
, pp. 140-149
-
-
Elbourne, D.R.1
Altman, D.G.2
Higgins, J.P.3
Curtin, F.4
Worthington, H.V.5
Vail, A.6
-
15
-
-
0041876133
-
Measuring inconsistency in meta-analysis
-
Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analysis. BMJ 2003;327(7414):555-7.
-
(2003)
BMJ
, vol.327
, Issue.7414
, pp. 555-557
-
-
Higgins, J.P.T.1
Thompson, S.G.2
Deeks, J.J.3
Altman, D.G.4
-
16
-
-
70049099036
-
Assessing risk of bias in included studies
-
In: Higgins JPT, Green S (editors) [updated March 2011] The Cochrane Collaboration
-
Higgins JPT, Altman DG. Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
-
(2011)
Cochrane Handbook for Systematic Reviews of Interventions Version 5.1
-
-
Higgins, J.P.T.1
Altman, D.G.2
-
17
-
-
84890613528
-
Special topics in statistics. Intention to treat issues.
-
In: Higgins JPT, Green S (editors) [updated March 2011] The Cochrane Collaboration
-
Higgins JPT, Deeks JJ, Altman DG. Chpater 16: Special topics in statistics. Intention to treat issues. In: Higgins JPT, Green S (editors). Cochrane Handbook for Systematic Reviews of Interventions Version 5.1 [updated March 2011]. The Cochrane Collaboration, 2011. Available from www.cochrane-handbook.org.
-
(2011)
Cochrane Handbook for Systematic Reviews of Interventions Version 5.1
-
-
Higgins, J.P.T.1
Deeks, J.J.2
Altman, D.G.3
-
18
-
-
69449091683
-
Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS)
-
Jones SA, Almássy Z, Beck M, Burt K, Clarke JT, Giugliani R, et al. Mortality and cause of death in mucopolysaccharidosis type II-a historical review based on data from the Hunter Outcome Survey (HOS). Journal of Inherited Metabolic Disease 2008;32(4):534-43.
-
(2008)
Journal of Inherited Metabolic Disease
, vol.32
, Issue.4
, pp. 534-543
-
-
Jones, S.A.1
Almássy, Z.2
Beck, M.3
Burt, K.4
Clarke, J.T.5
Giugliani, R.6
-
19
-
-
84876075883
-
The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS)
-
Jones SA, Parini R, Harmatz P, Giugliani R, Fang J, Mendelsohn NJ, et al. The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS). Molecular Genetics and Metabolism 2013;109(1):41-8.
-
(2013)
Molecular Genetics and Metabolism
, vol.109
, Issue.1
, pp. 41-48
-
-
Jones, S.A.1
Parini, R.2
Harmatz, P.3
Giugliani, R.4
Fang, J.5
Mendelsohn, N.J.6
-
20
-
-
0020009442
-
Enzymic diagnosis of the genetic mucopolysaccharide storage disorders
-
Kresse H, von Figura K, Klein U, Glossl J, Paschke E, Pohlmann R. Enzymic diagnosis of the genetic mucopolysaccharide storage disorders. Methods in Enzymology 1982;83:559-72.
-
(1982)
Methods in Enzymology
, vol.83
, pp. 559-572
-
-
Kresse, H.1
von Figura, K.2
Klein, U.3
Glossl, J.4
Paschke, E.5
Pohlmann, R.6
-
21
-
-
38849176942
-
Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)
-
Martin R, Beck M, Eng C, Giugliani R, Harmatz P, Munoz V, et al. Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome). Pediatrics 2008;121(2):e377-86.
-
(2008)
Pediatrics
, vol.121
, Issue.2
-
-
Martin, R.1
Beck, M.2
Eng, C.3
Giugliani, R.4
Harmatz, P.5
Munoz, V.6
-
22
-
-
0033585476
-
Prevalence of lysosomal storage disorders
-
Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA 1999;281(3):249-54.
-
(1999)
JAMA
, vol.281
, Issue.3
, pp. 249-254
-
-
Meikle, P.J.1
Hopwood, J.J.2
Clague, A.E.3
Carey, W.F.4
-
23
-
-
79951578486
-
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
-
Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, et al. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genetics in Medicine 2011;13(2):95-101.
-
(2011)
Genetics in Medicine
, vol.13
, Issue.2
, pp. 95-101
-
-
Muenzer, J.1
Beck, M.2
Eng, C.M.3
Giugliani, R.4
Harmatz, P.5
Martin, R.6
-
24
-
-
85041833980
-
New and Emerging Technology Briefing: Iduronate-2-sulfatase for Hunter Syndrome
-
National Horizon Scanning Centre
-
National Horizon Scanning Centre. New and Emerging Technology Briefing: Iduronate-2-sulfatase for Hunter Syndrome. The University of Birmingham 2005.
-
(2005)
The University of Birmingham
-
-
-
26
-
-
79955792706
-
Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II
-
Schulze-Frenking G, Jones SA, Roberts J, Beck M, Wraith JE. Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II. Journal of Inherited Metabolic Disease 2011;34(1):203-8.
-
(2011)
Journal of Inherited Metabolic Disease
, vol.34
, Issue.1
, pp. 203-208
-
-
Schulze-Frenking, G.1
Jones, S.A.2
Roberts, J.3
Beck, M.4
Wraith, J.E.5
-
27
-
-
15744375829
-
Mucopolysaccharidosis type II in females: case report and review of literature
-
Tuschl K, Gal A, Paschke E, Kircher S, Bodamer OA. Mucopolysaccharidosis type II in females: case report and review of literature. Pediatric Neurology 2005;32(4):270-2.
-
(2005)
Pediatric Neurology
, vol.32
, Issue.4
, pp. 270-272
-
-
Tuschl, K.1
Gal, A.2
Paschke, E.3
Kircher, S.4
Bodamer, O.A.5
-
28
-
-
79953845327
-
Guidelines for the investigation and management of patients with mucopolysaccharidosis type II
-
accessed September
-
Vellodi A, Wraith JE, Cleary M, Ramaswami U, Lavery C, Jessop E. Guidelines for the investigation and management of patients with mucopolysaccharidosis type II. www.specialisedservices.nhs.uk/library/23/Guidelines_for_Mucopolysaccharidosis_Type_II.pdf (accessed September 2009).
-
(2009)
-
-
Vellodi, A.1
Wraith, J.E.2
Cleary, M.3
Ramaswami, U.4
Lavery, C.5
Jessop, E.6
-
29
-
-
39149118050
-
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
-
Wraith JE, Scarpa M, Beck M, Bodamer OA, De Meirleir L, Guffon N, et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. European Journal of Pediatrics 2008;167(3):267-77.
-
(2008)
European Journal of Pediatrics
, vol.167
, Issue.3
, pp. 267-277
-
-
Wraith, J.E.1
Scarpa, M.2
Beck, M.3
Bodamer, O.A.4
De Meirleir, L.5
Guffon, N.6
-
30
-
-
41049096402
-
Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II
-
Wraith JE. Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II. Acta Paediatrica. Supplementum 2008;97(497):76-8.
-
(2008)
Acta Paediatrica. Supplementum
, vol.97
, Issue.497
, pp. 76-78
-
-
Wraith, J.E.1
-
31
-
-
0020613358
-
The natural history of the severe form of Hunter's syndrome: a study based on 52 cases
-
Young ID, Harper PS. The natural history of the severe form of Hunter's syndrome: a study based on 52 cases. Developmental Medicine and Child Neurology 1983;25(4):481-9.
-
(1983)
Developmental Medicine and Child Neurology
, vol.25
, Issue.4
, pp. 481-489
-
-
Young, I.D.1
Harper, P.S.2
-
32
-
-
84860803129
-
Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)
-
da Silva EMK, da Silva EMK, Strufaldi MWL, Andriolo RB, Silva LA. Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome). Cochrane Database of Systematic Reviews 2011, Issue 11. [DOI: ]
-
(2011)
Cochrane Database of Systematic Reviews
, Issue.11
-
-
da Silva, E.M.K.1
da Silva, E.M.K.2
Strufaldi, M.W.L.3
Andriolo, R.B.4
Silva, L.A.5
|