J Edmond Wraith

Summary

Affiliation: Manchester Royal Infirmary
Country: UK

Publications

  1. ncbi request reprint Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)
    James E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, United Kingdom
    J Pediatr 144:581-8. 2004
  2. ncbi request reprint The clinical presentation of lysosomal storage disorders
    James E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, UK
    Acta Neurol Taiwan 13:101-6. 2004
  3. ncbi request reprint The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I
    James Edward Wraith
    Royal Manchester Children s Hospital, Willink Biochemical Genetics Unit, Hospital Road, Manchester M27 4HA, UK
    Expert Opin Pharmacother 6:489-506. 2005
  4. pmc Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
    J Edmond Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Hospital Road, Manchester M27 4HA, UK
    Eur J Pediatr 167:267-77. 2008
  5. ncbi request reprint Enzyme replacement therapy for the management of the mucopolysaccharidoses
    J E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, UK
    Int J Clin Pharmacol Ther 47:S63-5. 2009
  6. pmc Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure
    Minke H de Ru
    Department of Paediatrics, Academic Medical Center, University Hospital of Amsterdam H7 270, Meibergdreef 9, 1105 AZ Amsterdam, The Netherlands
    Orphanet J Rare Dis 7:22. 2012
  7. pmc The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK
    David Moore
    Department of Public Health and Epidemiology, University of Birmingham, Birmingham, UK
    Orphanet J Rare Dis 3:24. 2008
  8. doi request reprint Initial report from the Hunter Outcome Survey
    J Edmond Wraith
    From the 1Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, United Kingdom
    Genet Med 10:508-16. 2008
  9. doi request reprint Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II
    J Edmond Wraith
    Royal Manchester Children s Hospital, Manchester, UK
    Acta Paediatr Suppl 97:76-8. 2008
  10. ncbi request reprint Limitations of enzyme replacement therapy: current and future
    J E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, M27 4HA, UK
    J Inherit Metab Dis 29:442-7. 2006

Collaborators

Detail Information

Publications16

  1. ncbi request reprint Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)
    James E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, United Kingdom
    J Pediatr 144:581-8. 2004
    ..To confirm the efficacy and safety of recombinant human alpha-L-iduronidase (laronidase) in patients with mucopolysaccharidosis I (MPS I)...
  2. ncbi request reprint The clinical presentation of lysosomal storage disorders
    James E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, UK
    Acta Neurol Taiwan 13:101-6. 2004
    ..g. Gaucher disease and Niemann-Pick B). Both urine and blood testing are necessary and the majority of diagnoses can now be confirmed at a molecular level. Prenatal diagnosis is possible for all...
  3. ncbi request reprint The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I
    James Edward Wraith
    Royal Manchester Children s Hospital, Willink Biochemical Genetics Unit, Hospital Road, Manchester M27 4HA, UK
    Expert Opin Pharmacother 6:489-506. 2005
    ..Results of the clinical trials and their initial extension periods, as well as the anecdotal experiences of physicians with laronidase in non-study settings, are discussed...
  4. pmc Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
    J Edmond Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Hospital Road, Manchester M27 4HA, UK
    Eur J Pediatr 167:267-77. 2008
    ..The issue of treating very young patients and those with CNS involvement is also discussed. ERT with idursulfase has the potential to benefit many patients with MPS II, especially if started early in the course of the disease...
  5. ncbi request reprint Enzyme replacement therapy for the management of the mucopolysaccharidoses
    J E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, UK
    Int J Clin Pharmacol Ther 47:S63-5. 2009
    ....
  6. pmc Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure
    Minke H de Ru
    Department of Paediatrics, Academic Medical Center, University Hospital of Amsterdam H7 270, Meibergdreef 9, 1105 AZ Amsterdam, The Netherlands
    Orphanet J Rare Dis 7:22. 2012
    ..Therefore, a numerical severity scale for classifying different MPS I phenotypes at diagnosis based on clinical signs and symptoms was developed...
  7. pmc The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK
    David Moore
    Department of Public Health and Epidemiology, University of Birmingham, Birmingham, UK
    Orphanet J Rare Dis 3:24. 2008
    ..In general such data on the natural history of lysosomal storage diseases is sparse...
  8. doi request reprint Initial report from the Hunter Outcome Survey
    J Edmond Wraith
    From the 1Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, United Kingdom
    Genet Med 10:508-16. 2008
    ..The Hunter Outcome Survey (HOS) was established to better describe the natural history of this disorder and to evaluate the long-term effect of enzyme replacement therapy...
  9. doi request reprint Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II
    J Edmond Wraith
    Royal Manchester Children s Hospital, Manchester, UK
    Acta Paediatr Suppl 97:76-8. 2008
    ....
  10. ncbi request reprint Limitations of enzyme replacement therapy: current and future
    J E Wraith
    Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, M27 4HA, UK
    J Inherit Metab Dis 29:442-7. 2006
    ..Finally, the therapies are expensive, limiting access to patients from those countries that are able to afford expensive health care...
  11. ncbi request reprint 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
    Royal Manchester Children s Hospital, Willink Biochemical Genetics Unit, Hospital Road, Pendlebury, Manchester M27 1HA, United Kingdom
    Pediatrics 120:e37-46. 2007
    ..Our objective was to evaluate the safety, pharmacokinetics, and efficacy of laronidase in young, severely affected children with mucopolysaccharidosis I...
  12. doi request reprint Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experience
    Daniel H Wiseman
    Department of Paediatric Haematology, Royal Manchester Children s Hospital, Manchester, UK
    J Inherit Metab Dis 36:263-70. 2013
    ....
  13. doi request reprint Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)
    Chris J Gardner
    Royal Manchester Children s Hospital, Manchester, UK
    J Inherit Metab Dis 34:489-97. 2011
    ..Regular screening for the development of endocrine sequelae is therefore essential. We present for the first time data on final adult height and endocrine complications in children with MPSIH post HSCT...
  14. doi request reprint Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients
    Robert F Wynn
    Blood and Marrow Transplant Unit, Royal Manchester Children s Hospital, Manchester, UK
    J Pediatr 154:135-9. 2009
    ..The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning...
  15. pmc Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease
    Ana Sergijenko
    Stem Cell and Neurotherapies, Institute of Human Development, Faculty of Medical and Human Sciences, University of Manchester, Manchester, UK
    Mol Ther 21:1938-49. 2013
    ..We demonstrate compelling evidence of neurological disease correction using autologous myeloid driven lentiviral-HSC gene therapy in MPSIIIA mice...
  16. doi request reprint Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry disease
    J Edmond Wraith
    Royal Manchester Children s Hospital, Manchester, United Kingdom
    J Pediatr 152:563-70, 570.e1. 2008
    ....