Research Topics
| J Edmond WraithSummaryAffiliation: Manchester Royal Infirmary Country: UK Publications
| Collaborators
|
Detail Information
Publications
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)...- The clinical presentation of lysosomal storage disordersJames 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... - The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis IJames 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... 
Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapyJ 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...- Enzyme replacement therapy for the management of the mucopolysaccharidosesJ E Wraith
Willink Biochemical Genetics Unit, Royal Manchester Children s Hospital, Manchester, UK
Int J Clin Pharmacol Ther 47:S63-5. 2009.... - Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedureMinke 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... - The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UKDavid 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... 
Initial report from the Hunter Outcome SurveyJ 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...- Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type IIJ Edmond Wraith
Royal Manchester Children s Hospital, Manchester, UK
Acta Paediatr Suppl 97:76-8. 2008..CONCLUSION: This short review provides an overview of the natural history of MPS II and current experience of enzyme replacement therapy with idursulfase... - 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... - Limitations of enzyme replacement therapy: current and futureJ 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... - 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... - Management of mucopolysaccharidosis type IH (Hurler's syndrome) presenting in infancy with severe dilated cardiomyopathy: a single institution's experienceDaniel H Wiseman
Department of Paediatric Haematology, Royal Manchester Children s Hospital, Manchester, UK
J Inherit Metab Dis 36:263-70. 2013.... - Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patientsRobert 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... - Safety and efficacy of enzyme replacement therapy with agalsidase beta: an international, open-label study in pediatric patients with Fabry diseaseJ Edmond Wraith
Royal Manchester Children s Hospital, Manchester, United Kingdom
J Pediatr 152:563-70, 570.e1. 2008....