Publications by authors named "Edmond Wraith"

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Haematopoietic Stem Cell Transplantation Arrests the Progression of Neurodegenerative Disease in Late-Onset Tay-Sachs Disease.
Karolina M Stepien, Su Han Lum, J Edmond Wraith, Christian J Hendriksz, Heather J Church

JIMD Rep· December 2017

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Assessment of musculoskeletal abnormalities in children with mucopolysaccharidoses using pGALS.
Mercedes O Chan, Ethan S Sen, Elizabeth Hardy, Pauline Hensman, Edmond Wraith

Pediatr Rheumatol Online J· May 2015

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Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.
H Angharad Watson, Rebecca J Holley, Kia J Langford-Smith, Fiona L Wilkinson, Toin H van Kuppevelt, J Edmond Wraith

J Biol Chem· December 2014

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Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease.
Ana Sergijenko, Alexander Langford-Smith, Ai Y Liao, Claire E Pickford, John McDermott, J Edmond Wraith

Mol Ther· October 2013

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Clinical effect and safety profile of recombinant human lysosomal acid lipase in patients with cholesteryl ester storage disease.
Manisha Balwani, Catherine Breen, Gregory M Enns, Patrick B Deegan, Tomas Honzík, J Edmond Wraith

Hepatology· September 2013

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Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience.
Daniel H Wiseman, Jean Mercer, Karen Tylee, Nilima Malaiya, Denise K Bonney, J Edmond Wraith

J Inherit Metab Dis· March 2013

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Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure.
Minke H de Ru, Quirine Ga Teunissen, Johanna H van der Lee, Michael Beck, Olaf A Bodamer, J Edmond Wraith

Orphanet J Rare Dis· April 2012

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High rate of postoperative mortality in patients with mucopolysaccharidosis I: findings from the MPS I Registry.
Pamela Arn, Chester Whitley, J Edmond Wraith, H Warner Webb, Lisa Underhill

J Pediatr Surg· March 2012

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The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome).
Elke Miebach, Heather Church, Alan Cooper, Jean Mercer, Karen Tylee, J Edmond Wraith

J Inherit Metab Dis· June 2011

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Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H).
Chris J Gardner, Nicola Robinson, Tim Meadows, Robert Wynn, Andrew Will, J Edmond Wraith

J Inherit Metab Dis· April 2011

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Potential biomarkers of osteonecrosis in Gaucher disease.
Elena V Pavlova, Patrick B Deegan, Jane Tindall, Ian McFarlane, Atul Mehta, J Edmond Wraith

Blood Cells Mol Dis· January 2011

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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.
Paul Harmatz, Zi-Fan Yu, Roberto Giugliani, Ida Vanessa D Schwartz, Nathalie Guffon, J Edmond Wraith

J Inherit Metab Dis· February 2010

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Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase.
Celeste Decker, Zi-Fan Yu, Roberto Giugliani, Ida Vanessa D Schwartz, Nathalie Guffon, J Edmond Wraith

J Pediatr Rehabil Med· January 2010

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Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry.
Pamela Arn, J Edmond Wraith, Lisa Underhill

J Pediatr· June 2009

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Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
Lorne A Clarke, J Edmond Wraith, Michael Beck, Edwin H Kolodny, Gregory M Pastores

Pediatrics· January 2009

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Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients.
Robert F Wynn, Jean Mercer, Joanne Page, Trevor F Carr, Simon Jones, J Edmond Wraith

J Pediatr· January 2009

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Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
Paul Harmatz, Roberto Giugliani, Ida Vanessa D Schwartz, Nathalie Guffon, Elisa Leão Teles, J Edmond Wraith

Mol Genet Metab· August 2008

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Initial report from the Hunter Outcome Survey.
J Edmond Wraith, Michael Beck, Roberto Giugliani, Joe Clarke, Rick Martin

Genet Med· July 2008

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Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease.
J Edmond Wraith, Anna Tylki-Szymanska, Nathalie Guffon, Y Howard Lien, Michel Tsimaratos

J Pediatr· April 2008

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Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II.
J Edmond Wraith

Acta Paediatr· April 2008

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Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy.
J Edmond Wraith, Maurizio Scarpa, Michael Beck, Olaf A Bodamer, Linda De Meirleir

Eur J Pediatr· March 2008

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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).
J Edmond Wraith, Michael Beck, Roderick Lane, Ans van der Ploeg, Elsa Shapiro

Pediatrics· July 2007

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The MPS I registry: design, methodology, and early findings of a global disease registry for monitoring patients with Mucopolysaccharidosis Type I.
Gregory M Pastores, Pamela Arn, Michael Beck, Joe T R Clarke, Nathalie Guffon, J Edmond Wraith

Mol Genet Metab· May 2007

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Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study.
Paul Harmatz, Roberto Giugliani, Ida Schwartz, Nathalie Guffon, Elisa Leão Teles, J Edmond Wraith

J Pediatr· April 2006

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