-
1
-
-
0015798495
-
The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase
-
Bach G., Eisenberg F., Cantz M., Neufeld E.F. The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. Proc. Natl. Acad. Sci. U. S. A. 1973, 70:2134-2138.
-
(1973)
Proc. Natl. Acad. Sci. U. S. A.
, vol.70
, pp. 2134-2138
-
-
Bach, G.1
Eisenberg, F.2
Cantz, M.3
Neufeld, E.F.4
-
2
-
-
38849176942
-
Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
-
Martin R., Beck M., Eng C., Giugliani R., Harmatz P., Munoz V., Muenzer J. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 2008, 121:e377-e386.
-
(2008)
Pediatrics
, vol.121
-
-
Martin, R.1
Beck, M.2
Eng, C.3
Giugliani, R.4
Harmatz, P.5
Munoz, V.6
Muenzer, J.7
-
3
-
-
33747209013
-
A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)
-
Muenzer J., Wraith J.E., Beck M., Giugliani R., Harmatz P., Eng C.M., Vellodi A., Martin R., Ramaswami U., Gucsavas-Calikoglu M., Vijayaraghavan S., Wendt S., Puga A.C., Ulbrich B., Shinawi M., Cleary M., Piper D., Conway A.M., Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet. Med. 2006, 8:465-473.
-
(2006)
Genet. Med.
, vol.8
, pp. 465-473
-
-
Muenzer, J.1
Wraith, J.E.2
Beck, M.3
Giugliani, R.4
Harmatz, P.5
Eng, C.M.6
Vellodi, A.7
Martin, R.8
Ramaswami, U.9
Gucsavas-Calikoglu, M.10
Vijayaraghavan, S.11
Wendt, S.12
Puga, A.C.13
Ulbrich, B.14
Shinawi, M.15
Cleary, M.16
Piper, D.17
Conway, A.M.18
Kimura, A.19
-
4
-
-
71649090838
-
Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)
-
Okuyama T., Tanaka A., Suzuki Y., Ida H., Tanaka T., Cox G.F., Eto Y., Orii T. Japan Elaprase Treatment (JET) study: idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II). Mol. Genet. Metab. 2010, 99:18-25.
-
(2010)
Mol. Genet. Metab.
, vol.99
, pp. 18-25
-
-
Okuyama, T.1
Tanaka, A.2
Suzuki, Y.3
Ida, H.4
Tanaka, T.5
Cox, G.F.6
Eto, Y.7
Orii, T.8
-
5
-
-
79951578486
-
Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome
-
Muenzer J., Beck M., Eng C.M., Giugliani R., Harmatz P., Martin R., Ramaswami U., Vellodi A., Wraith J.E., Cleary M., Gucsavas-Calikoglu M., Puga A.C., Shinawi M., Ulbrich B., Vijayaraghavan S., Wendt S., Conway A.M., Rossi A., Whiteman D.A., Kimura A. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genet. Med. 2011, 13:95-101.
-
(2011)
Genet. Med.
, vol.13
, pp. 95-101
-
-
Muenzer, J.1
Beck, M.2
Eng, C.M.3
Giugliani, R.4
Harmatz, P.5
Martin, R.6
Ramaswami, U.7
Vellodi, A.8
Wraith, J.E.9
Cleary, M.10
Gucsavas-Calikoglu, M.11
Puga, A.C.12
Shinawi, M.13
Ulbrich, B.14
Vijayaraghavan, S.15
Wendt, S.16
Conway, A.M.17
Rossi, A.18
Whiteman, D.A.19
Kimura, A.20
more..
-
6
-
-
39149118050
-
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
-
Wraith J.E., Scarpa M., Beck M., Bodamer O.A., De Meirleir L., Guffon N., Meldgaard Lund A., Malm G., Van der Ploeg A.T., Zeman J. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur. J. Pediatr. 2008, 167:267-277.
-
(2008)
Eur. J. Pediatr.
, vol.167
, pp. 267-277
-
-
Wraith, J.E.1
Scarpa, M.2
Beck, M.3
Bodamer, O.A.4
De Meirleir, L.5
Guffon, N.6
Meldgaard Lund, A.7
Malm, G.8
Van der Ploeg, A.T.9
Zeman, J.10
-
7
-
-
50149108962
-
Neurological findings in Hunter disease: pathology and possible therapeutic effects reviewed
-
Al Sawaf S., Mayatepek E., Hoffmann B. Neurological findings in Hunter disease: pathology and possible therapeutic effects reviewed. J. Inherit. Metab. Dis. 2008, 31:473-480.
-
(2008)
J. Inherit. Metab. Dis.
, vol.31
, pp. 473-480
-
-
Al Sawaf, S.1
Mayatepek, E.2
Hoffmann, B.3
-
8
-
-
84889593058
-
Massive accumulation of glycosaminoglycans in the aortic valve of a patient with Hunter syndrome during enzyme replacement therapy
-
(in press)
-
Sato Y., Fujiwara M., Kobayashi H., Ida H. Massive accumulation of glycosaminoglycans in the aortic valve of a patient with Hunter syndrome during enzyme replacement therapy. Pediatr. Cardiol. 2013, (in press).
-
(2013)
Pediatr. Cardiol.
-
-
Sato, Y.1
Fujiwara, M.2
Kobayashi, H.3
Ida, H.4
-
9
-
-
84868272640
-
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. The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders. Health Technol. Assess. 2012, 16:1-543.
-
(2012)
Health Technol. Assess.
, vol.16
, pp. 1-543
-
-
Wyatt, K.1
Henley, W.2
Anderson, L.3
Anderson, R.4
Nikolaou, V.5
Stein, K.6
Klinger, L.7
Hughes, D.8
Waldek, S.9
Lachmann, R.10
Mehta, A.11
Vellodi, A.12
Logan, S.13
-
10
-
-
0037295890
-
Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines
-
National Marrow Donor Program International Bone Marrow Transplant Registry Working Party on Inborn Errors, European Bone Marrow Transplant Group
-
Peters C., Steward C.G., National Marrow Donor Program, International Bone Marrow Transplant Registry, Working Party on Inborn Errors, European Bone Marrow Transplant Group Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant. 2003, 31:229-239.
-
(2003)
Bone Marrow Transplant.
, vol.31
, pp. 229-239
-
-
Peters, C.1
Steward, C.G.2
-
11
-
-
33745099961
-
Trends in haematopoietic cell transplantation for inborn errors of metabolism
-
Boelens J.J. Trends in haematopoietic cell transplantation for inborn errors of metabolism. J. Inherit. Metab. Dis. 2006, 29:413-420.
-
(2006)
J. Inherit. Metab. Dis.
, vol.29
, pp. 413-420
-
-
Boelens, J.J.1
-
12
-
-
0029146242
-
Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation
-
Shapiro E.G., Lockman L.A., Balthazor M., Krivit W. Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J. Inherit. Metab. Dis. 1995, 18:413-429.
-
(1995)
J. Inherit. Metab. Dis.
, vol.18
, pp. 413-429
-
-
Shapiro, E.G.1
Lockman, L.A.2
Balthazor, M.3
Krivit, W.4
-
13
-
-
0029795037
-
Bone marrow transplantation in Hunter syndrome
-
McKinnis E.J., Sulzbacher S., Rutledge J.C., Sanders J., Scott C.R. Bone marrow transplantation in Hunter syndrome. J. Pediatr. 1996, 129:145-148.
-
(1996)
J. Pediatr.
, vol.129
, pp. 145-148
-
-
McKinnis, E.J.1
Sulzbacher, S.2
Rutledge, J.C.3
Sanders, J.4
Scott, C.R.5
-
14
-
-
0032998144
-
Long-term follow-up following bone marrow transplantation for Hunter disease
-
Vellodi A., Young E., Cooper A., Lidchi V., Winchester B., Wraith J.E. Long-term follow-up following bone marrow transplantation for Hunter disease. J. Inherit. Metab. Dis. 1999, 22:638-648.
-
(1999)
J. Inherit. Metab. Dis.
, vol.22
, pp. 638-648
-
-
Vellodi, A.1
Young, E.2
Cooper, A.3
Lidchi, V.4
Winchester, B.5
Wraith, J.E.6
-
15
-
-
64549141555
-
Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17years
-
Guffon N., Bertrand Y., Forest I., Fouilhoux A., Froissart R. Bone marrow transplantation in children with Hunter syndrome: outcome after 7 to 17years. J. Pediatr. 2009, 154:733-737.
-
(2009)
J. Pediatr.
, vol.154
, pp. 733-737
-
-
Guffon, N.1
Bertrand, Y.2
Forest, I.3
Fouilhoux, A.4
Froissart, R.5
-
16
-
-
84867901875
-
Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan
-
Tanaka A., Okuyama T., Suzuki Y., Sakai N., Takakura H., Sawada T., Tanaka T., Otomo T., Ohashi T., Ishige-Wada M., Yabe H., Ohura T., Suzuki N., Kato K., Adachi S., Kobayashi R., Mugishima H., Kato S. Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: a nationwide survey in Japan. Mol. Genet. Metab. 2012, 107:513-520.
-
(2012)
Mol. Genet. Metab.
, vol.107
, pp. 513-520
-
-
Tanaka, A.1
Okuyama, T.2
Suzuki, Y.3
Sakai, N.4
Takakura, H.5
Sawada, T.6
Tanaka, T.7
Otomo, T.8
Ohashi, T.9
Ishige-Wada, M.10
Yabe, H.11
Ohura, T.12
Suzuki, N.13
Kato, K.14
Adachi, S.15
Kobayashi, R.16
Mugishima, H.17
Kato, S.18
-
17
-
-
37249043762
-
The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome)
-
Garcia A.R., Pan J., Lamsa J.C., Muenzer J. The characterization of a murine model of mucopolysaccharidosis II (Hunter syndrome). J. Inherit. Metab. Dis. 2007, 30:924-934.
-
(2007)
J. Inherit. Metab. Dis.
, vol.30
, pp. 924-934
-
-
Garcia, A.R.1
Pan, J.2
Lamsa, J.C.3
Muenzer, J.4
-
18
-
-
79958088297
-
Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease
-
Yokoi T., Kobayashi H., Shimada Y., Eto Y., Ishige N., Kitagawa T., Otsu M., Nakauchi H., Ida H., Ohashi T. Minimum requirement of donor cells to reduce the glycolipid storage following bone marrow transplantation in a murine model of Fabry disease. J. Gene Med. 2011, 13:262-268.
-
(2011)
J. Gene Med.
, vol.13
, pp. 262-268
-
-
Yokoi, T.1
Kobayashi, H.2
Shimada, Y.3
Eto, Y.4
Ishige, N.5
Kitagawa, T.6
Otsu, M.7
Nakauchi, H.8
Ida, H.9
Ohashi, T.10
-
19
-
-
84866171251
-
Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II
-
Higuchi T., Shimizu H., Fukuda T., Kawagoe S., Matsumoto J., Shimada Y., Kobayashi H., Ida H., Ohashi T., Morimoto H., Hirato T., Nishino K., Eto Y. Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II. Mol. Genet. Metab. 2012, 107:122-128.
-
(2012)
Mol. Genet. Metab.
, vol.107
, pp. 122-128
-
-
Higuchi, T.1
Shimizu, H.2
Fukuda, T.3
Kawagoe, S.4
Matsumoto, J.5
Shimada, Y.6
Kobayashi, H.7
Ida, H.8
Ohashi, T.9
Morimoto, H.10
Hirato, T.11
Nishino, K.12
Eto, Y.13
-
20
-
-
84862777079
-
Disease-specific non-reducing end carbohydrate biomarkers for mucopolysaccharidoses
-
Lawrence R., Brown J.R., Al-Mafraji K., Lamanna W.C., Beitel J.R., Boons G.J., Esko J.D., Crawford B.E. Disease-specific non-reducing end carbohydrate biomarkers for mucopolysaccharidoses. Nat. Chem. Biol. 2012, 8:197-204.
-
(2012)
Nat. Chem. Biol.
, vol.8
, pp. 197-204
-
-
Lawrence, R.1
Brown, J.R.2
Al-Mafraji, K.3
Lamanna, W.C.4
Beitel, J.R.5
Boons, G.J.6
Esko, J.D.7
Crawford, B.E.8
-
21
-
-
79551601711
-
Growth pattern and growth prediction of body height in children with mucopolysaccharidosis type II
-
Rozdzynska A., Tylki-Szymanska A., Jurecka A., Cieslik J. Growth pattern and growth prediction of body height in children with mucopolysaccharidosis type II. Acta Paediatr. 2011, 100:456-460.
-
(2011)
Acta Paediatr.
, vol.100
, pp. 456-460
-
-
Rozdzynska, A.1
Tylki-Szymanska, A.2
Jurecka, A.3
Cieslik, J.4
-
22
-
-
84866984376
-
Administration of anti-CD3 antibodies modulates the immune response to an infusion of alpha-glucosidase in mice
-
Ohashi T., Iizuka S., Shimada Y., Higuchi T., Eto Y., Ida H., Kobayashi H. Administration of anti-CD3 antibodies modulates the immune response to an infusion of alpha-glucosidase in mice. Mol. Ther. 2012, 20:1924-1931.
-
(2012)
Mol. Ther.
, vol.20
, pp. 1924-1931
-
-
Ohashi, T.1
Iizuka, S.2
Shimada, Y.3
Higuchi, T.4
Eto, Y.5
Ida, H.6
Kobayashi, H.7
-
23
-
-
0019466750
-
Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation
-
Hobbs J.R., Hugh-Jones K., Barrett A.J., Byrom N., Chambers D., Henry K., James D.C., Lucas C.F., Rogers T.R., Benson P.F., Tansley L.R., Patrick A.D., Mossman J., Young E.P. Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981, 2:709-712.
-
(1981)
Lancet
, vol.2
, pp. 709-712
-
-
Hobbs, J.R.1
Hugh-Jones, K.2
Barrett, A.J.3
Byrom, N.4
Chambers, D.5
Henry, K.6
James, D.C.7
Lucas, C.F.8
Rogers, T.R.9
Benson, P.F.10
Tansley, L.R.11
Patrick, A.D.12
Mossman, J.13
Young, E.P.14
-
24
-
-
59449100963
-
Mucopolysaccharidosis I: management and treatment guidelines
-
International Consensus Panel on Management and Treatment of Mucopolysaccharidosis
-
Muenzer J., Wraith J.E., Clarke L.A., International Consensus Panel on Management and Treatment of Mucopolysaccharidosis Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics 2009, 123:19-29.
-
(2009)
Pediatrics
, vol.123
, pp. 19-29
-
-
Muenzer, J.1
Wraith, J.E.2
Clarke, L.A.3
-
25
-
-
84865976292
-
Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I
-
Wolf D.A., Lenander A.W., Nan Z., Braunlin E.A., Podetz-Pedersen K.M., Whitley C.B., Gupta P., Low W.C., McIvor R.S. Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I. Bone Marrow Transplant. 2012, 47:1235-1240.
-
(2012)
Bone Marrow Transplant.
, vol.47
, pp. 1235-1240
-
-
Wolf, D.A.1
Lenander, A.W.2
Nan, Z.3
Braunlin, E.A.4
Podetz-Pedersen, K.M.5
Whitley, C.B.6
Gupta, P.7
Low, W.C.8
McIvor, R.S.9
-
26
-
-
77956428075
-
Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice
-
Lau A.A., Hannouche H., Rozaklis T., Hassiotis S., Hopwood J.J., Hemsley K.M. Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice. Exp. Neurol. 2010, 225:445-454.
-
(2010)
Exp. Neurol.
, vol.225
, pp. 445-454
-
-
Lau, A.A.1
Hannouche, H.2
Rozaklis, T.3
Hassiotis, S.4
Hopwood, J.J.5
Hemsley, K.M.6
-
27
-
-
84864564603
-
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice
-
Langford-Smith A., Wilkinson F.L., Langford-Smith K.J., Holley R.J., Sergijenko A., Howe S.J., Bennett W.R., Jones S.A., Wraith J., Merry C.L., Wynn R.F., Bigger B.W. Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. Mol. Ther. 2012, 20:1610-1621.
-
(2012)
Mol. Ther.
, vol.20
, pp. 1610-1621
-
-
Langford-Smith, A.1
Wilkinson, F.L.2
Langford-Smith, K.J.3
Holley, R.J.4
Sergijenko, A.5
Howe, S.J.6
Bennett, W.R.7
Jones, S.A.8
Wraith, J.9
Merry, C.L.10
Wynn, R.F.11
Bigger, B.W.12
-
28
-
-
71649099089
-
Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
-
Kishnani P.S., Goldenberg P.C., DeArmey S.L., Heller J., Benjamin D., Young S., Bali D., Smith S.A., Li J.S., Mandel H., Koeberl D., Rosenberg A., Chen Y.T. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol. Genet. Metab. 2010, 99:26-33.
-
(2010)
Mol. Genet. Metab.
, vol.99
, pp. 26-33
-
-
Kishnani, P.S.1
Goldenberg, P.C.2
DeArmey, S.L.3
Heller, J.4
Benjamin, D.5
Young, S.6
Bali, D.7
Smith, S.A.8
Li, J.S.9
Mandel, H.10
Koeberl, D.11
Rosenberg, A.12
Chen, Y.T.13
-
29
-
-
80051799963
-
The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease
-
Banugaria S.G., Prater S.N., Ng Y.K., Kobori J.A., Finkel R.S., Ladda R.L., Chen Y.T., Rosenberg A.S., Kishnani P.S. The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. Genet. Med. 2011, 13:729-736.
-
(2011)
Genet. Med.
, vol.13
, pp. 729-736
-
-
Banugaria, S.G.1
Prater, S.N.2
Ng, Y.K.3
Kobori, J.A.4
Finkel, R.S.5
Ladda, R.L.6
Chen, Y.T.7
Rosenberg, A.S.8
Kishnani, P.S.9
-
30
-
-
78649323110
-
Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): a single dose of galsulfase further reduces urine glycosaminoglycans after hematopoietic stem cell transplantation
-
Whitley C.B., Utz J.R. Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI): a single dose of galsulfase further reduces urine glycosaminoglycans after hematopoietic stem cell transplantation. Mol. Genet. Metab. 2010, 101:346-348.
-
(2010)
Mol. Genet. Metab.
, vol.101
, pp. 346-348
-
-
Whitley, C.B.1
Utz, J.R.2
-
31
-
-
84859973875
-
Enzyme replacement therapy improves joint motion and outcome of the 12-min walk test in a mucopolysaccharidosis type VI patient previously treated with bone marrow transplantation
-
Sohn Y.B., Park S.W., Kim S.H., Cho S.Y., Ji S.T., Kwon E.K., Han S.J., Oh S.J., Park Y.J., Ko A.R., Paik K.H., Lee J., Lee D.H., Jin D.K. Enzyme replacement therapy improves joint motion and outcome of the 12-min walk test in a mucopolysaccharidosis type VI patient previously treated with bone marrow transplantation. Am. J. Med. Genet. A 2012, 58A:1158-1163.
-
(2012)
Am. J. Med. Genet. A
, vol.58 A
, pp. 1158-1163
-
-
Sohn, Y.B.1
Park, S.W.2
Kim, S.H.3
Cho, S.Y.4
Ji, S.T.5
Kwon, E.K.6
Han, S.J.7
Oh, S.J.8
Park, Y.J.9
Ko, A.R.10
Paik, K.H.11
Lee, J.12
Lee, D.H.13
Jin, D.K.14
-
32
-
-
20944443759
-
Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome
-
Grewal S.S., Wynn R., Abdenur J.E., Burton B.K., Gharib M., Haase C., Hayashi R.J., Shenoy S., Sillence D., Tiller G.E., Dudek M.E., van Royen-Kerkhof A., Wraith J.E., Woodard P., Young G.A., Wulffraat N., Whitley C.B., Peters C. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet. Med. 2005, 7:143-146.
-
(2005)
Genet. Med.
, vol.7
, pp. 143-146
-
-
Grewal, S.S.1
Wynn, R.2
Abdenur, J.E.3
Burton, B.K.4
Gharib, M.5
Haase, C.6
Hayashi, R.J.7
Shenoy, S.8
Sillence, D.9
Tiller, G.E.10
Dudek, M.E.11
van Royen-Kerkhof, A.12
Wraith, J.E.13
Woodard, P.14
Young, G.A.15
Wulffraat, N.16
Whitley, C.B.17
Peters, C.18
-
33
-
-
33745257246
-
Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome
-
Cox-Brinkman J., Boelens J.J., Wraith J.E., O'meara A., Veys P., Wijburg F.A., Wulffraat N., Wynn R.F. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant. 2006, 38:17-21.
-
(2006)
Bone Marrow Transplant.
, vol.38
, pp. 17-21
-
-
Cox-Brinkman, J.1
Boelens, J.J.2
Wraith, J.E.3
O'meara, A.4
Veys, P.5
Wijburg, F.A.6
Wulffraat, N.7
Wynn, R.F.8
-
34
-
-
41549136110
-
Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome
-
Tolar J., Grewal S.S., Bjoraker K.J., Whitley C.B., Shapiro E.G., Charnas L., Orchard P.J. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome. Bone Marrow Transplant. 2008, 41:531-535.
-
(2008)
Bone Marrow Transplant.
, vol.41
, pp. 531-535
-
-
Tolar, J.1
Grewal, S.S.2
Bjoraker, K.J.3
Whitley, C.B.4
Shapiro, E.G.5
Charnas, L.6
Orchard, P.J.7
-
35
-
-
57249094012
-
Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients
-
Wynn R.F., Mercer J., Page J., Carr T.F., Jones S., Wraith J.E. Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. J. Pediatr. 2009, 154:135-139.
-
(2009)
J. Pediatr.
, vol.154
, pp. 135-139
-
-
Wynn, R.F.1
Mercer, J.2
Page, J.3
Carr, T.F.4
Jones, S.5
Wraith, J.E.6
-
36
-
-
85040148540
-
Combined enzyme replacement therapy and hematopoietic stem cell transplantation in mucopolysaccharidosis type VI
-
Sillence D., Waters K., Donaldson S., Shaw P.J., Ellaway C. Combined enzyme replacement therapy and hematopoietic stem cell transplantation in mucopolysaccharidosis type VI. JIMD Rep. 2012, 2:103-106.
-
(2012)
JIMD Rep.
, vol.2
, pp. 103-106
-
-
Sillence, D.1
Waters, K.2
Donaldson, S.3
Shaw, P.J.4
Ellaway, C.5
-
37
-
-
84862805856
-
Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I
-
Dickson P.I., Ellinwood N.M., Brown J.R., Witt R.G., Le S.Q., Passage M.B., Vera M.U., Crawford B.E. Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I. Mol. Genet. Metab. 2012, 106:68-72.
-
(2012)
Mol. Genet. Metab.
, vol.106
, pp. 68-72
-
-
Dickson, P.I.1
Ellinwood, N.M.2
Brown, J.R.3
Witt, R.G.4
Le, S.Q.5
Passage, M.B.6
Vera, M.U.7
Crawford, B.E.8
-
38
-
-
84893709837
-
Glycan-based biomarkers for mucopolysaccharidoses
-
Lawrence R., Brown J.R., Lorey F., Dickson P.I., Crawford B.E., Esko J.D. Glycan-based biomarkers for mucopolysaccharidoses. Mol. Genet. Metab. 2013, 111:73-83.
-
(2013)
Mol. Genet. Metab.
, vol.111
, pp. 73-83
-
-
Lawrence, R.1
Brown, J.R.2
Lorey, F.3
Dickson, P.I.4
Crawford, B.E.5
Esko, J.D.6
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