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ArticlesPeer ReviewedFull Text
Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease
Author / Creator:Sergijenko, Ana ; Langford-Smith, Alexander ; Liao, Ai Y ; Pickford, Claire E ; McDermott, John ; Nowinski, Gabriel ; Langford-Smith, Kia J ; Merry, Catherine LR ; Jones, Simon A ; Wraith, J Edmond ; Wynn, Robert F ; Wilkinson, Fiona L ; Bigger, Brian W
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2013-10, Vol.21 (10), p.1938-1949
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ArticlesPeer ReviewedFull Text
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
Author / Creator:Wraith, J. Edmond ; Scarpa, Maurizio ; Beck, Michael ; Bodamer, Olaf A. ; De Meirleir, Linda ; Guffon, Nathalie ; Meldgaard Lund, Allan ; Malm, Gunilla ; Van der Ploeg, Ans T. ; Zeman, Jiri
Publisher:Berlin/Heidelberg: Springer-Verlag
Journal title:European journal of pediatrics, 2008-03, Vol.167 (3), p.267-277
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ArticlesPeer ReviewedFull Text
Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase
Author / Creator:Harmatz, Paul ; Yu, Zi-Fan ; Giugliani, Roberto ; Schwartz, Ida Vanessa D ; Guffon, Nathalie ; Teles, Elisa Leão ; Miranda, M. Clara Sá ; Wraith, J. Edmond ; Beck, Michael ; Arash, Laila ; Scarpa, Maurizio ; Ketteridge, David ; Hopwood, John J ; Plecko, Barbara ; Steiner, Robert ; Whitley, Chester B ; Kaplan, Paige ; Swiedler, Stuart J ; Hardy, Karen ; Berger, Kenneth I ; Decker, Celeste
Publisher:Dordrecht: Dordrecht : Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2010-02, Vol.33 (1), p.51-60
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ArticlesPeer ReviewedFull Text
Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure
Author / Creator:de Ru, Minke H ; Teunissen, Quirine GA ; van der Lee, Johanna H ; Beck, Michael ; Bodamer, Olaf A ; Clarke, Lorne A ; Hollak, Carla E ; Lin, Shuan-Pei ; Rojas, Maria-Verónica Muñoz ; Pastores, Gregory M ; Raiman, Julian A ; Scarpa, Maurizio ; Treacy, Eileen P ; Tylki-Szymanska, Anna ; Edmond Wraith, J ; Zeman, Jiri ; Wijburg, Frits A
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2012-04, Vol.7 (1), p.22-22, Article 22
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ArticlesPeer ReviewedFull Text
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)
Author / Creator:Gardner, Chris J. ; Robinson, Nicola ; Meadows, Tim ; Wynn, Robert ; Will, Andrew ; Mercer, Jean ; Church, Heather J. ; Tylee, Karen ; Wraith, J. Edmond ; Clayton, Peter E.
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2011-04, Vol.34 (2), p.489-497
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ArticlesPeer ReviewedFull Text
Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human α- l -Iduronidase (Laronidase)
Author / Creator:Wraith, J. Edmond ; Beck, Michael ; Lane, Roderick ; van der Ploeg, Ans ; Shapiro, Elsa ; Xue, Yong ; Kakkis, Emil D. ; Guffon, Nathalie
Publisher:United States
Journal title:Pediatrics (Evanston), 2007-07, Vol.120 (1), p.e37-e46
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ArticlesPeer ReviewedFull Text
Management of mucopolysaccharidosis type IH (Hurler’s syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution’s experience
Author / Creator:Wiseman, Daniel H. ; Mercer, Jean ; Tylee, Karen ; Malaiya, Nilima ; Bonney, Denise K. ; Jones, Simon A. ; Wraith, J Edmond ; Wynn, Robert F.
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2013-03, Vol.36 (2), p.263-270
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ArticlesPeer ReviewedFull Text
The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome)
Author / Creator:Miebach, Elke ; Church, Heather ; Cooper, Alan ; Mercer, Jean ; Tylee, Karen ; Wynn, Robert F. ; Wraith, J. Edmond
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2011-06, Vol.34 (3), p.755-761
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ArticlesPeer ReviewedFull Text
Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human {alpha}-L-Iduronidase (Laronidase)
Author / Creator:Wraith, J. Edmond ; Beck, Michael ; Lane, Roderick ; van der Ploeg, Ans ; Shapiro, Elsa ; Xue, Yong ; Kakkis, Emil D ; Guffon, Nathalie
Publisher:Evanston: Am Acad Pediatrics
Journal title:Pediatrics (Evanston), 2007-07, Vol.120 (1), p.e37
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ArticlesPeer ReviewedFull Text
Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience: Mucopolysaccharidoses
Author / Creator:WISEMAN, Daniel H ; MERCER, Jean ; TYLEE, Karen ; MALAIYA, Nilima ; BONNEY, Denise K ; JONES, Simon A ; WRAITH, J. Edmond ; WYNN, Robert F
Publisher:Heidelberg: Springer
Journal title:Journal of inherited metabolic disease, 2013, Vol.36 (2), p.263-270
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ArticlesPeer ReviewedFull Text
Idursulfase for enzyme-replacement therapy in mucopolysaccharidosis II
Author / Creator:Wraith, J Edmond
Publisher:London: Future Medicine Ltd
Journal title:Clinical practice (London, England), 2007-05, Vol.4 (3), p.231-240
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ArticlesPeer ReviewedFull Text
Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)
Author / Creator:Hendriksz, Christian J. ; Giugliani, Roberto ; Harmatz, Paul ; Lampe, Christina ; Martins, Ana Maria ; Pastores, Gregory M. ; Steiner, Robert D. ; Leão Teles, Elisa ; Valayannopoulos, Vassili
Publisher:Dordrecht: Springer Netherlands
Journal title:Journal of inherited metabolic disease, 2013-03, Vol.36 (2), p.373-384
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Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

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Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

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Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

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Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure

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Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)

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Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human α- l -Iduronidase (Laronidase)

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Management of mucopolysaccharidosis type IH (Hurler’s syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution’s experience

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The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome)

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Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human {alpha}-L-Iduronidase (Laronidase)

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Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience: Mucopolysaccharidoses

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Idursulfase for enzyme-replacement therapy in mucopolysaccharidosis II

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Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP)

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