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Acta Ophthalmologica
|
December 15, 2017
The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler)
Ahmed Javed, Tariq Aslam, Simon A Jones, et al.
Journal of Inherited Metabolic Disease
|
June 22, 2012
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, et al.
Human Mutation
|
July 29, 2017
IDUA mutational profile and genotype-phenotype relationships in UK patients with Mucopolysaccharidosis Type I
Arunabha Ghosh, Jean Mercer, Sabrina Mackinnon, et al.
Journal of Inherited Metabolic Disease
|
January 22, 2011
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)
Chris J Gardner, Nicola Robinson, Tim Meadows, et al.
Orphanet Journal of Rare Diseases
|
January 22, 2021
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England
Maureen Cleary, James Davison, Rachel Gould, et al.
Journal of Inherited Metabolic Disease
|
December 21, 2010
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases
Kia Jane Langford-Smith, Jean Mercer, June Petty, et al.
Molecular Genetics and Metabolism
|
February 3, 2016
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres
Arunabha Ghosh, Weston Miller, Paul J Orchard, et al.
Haematologica
|
February 29, 2012
Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy
Muhammad Ameer Saif, Brian W Bigger, Karen E Brookes, et al.
Orphanet Journal of Rare Diseases
|
April 19, 2015
Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome
Abhijit Ricky Pal, Eveline J Langereis, Muhammad A Saif, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
|
February 25, 2015
Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines
Mieke Aldenhoven, Simon A Jones, Denise Bonney, et al.
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Search research articles
Search
Showing results (21-30 of 35) with videos related to
Sort By:
Page
of 4
Acta Ophthalmologica
|
December 15, 2017
The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler)
Ahmed Javed, Tariq Aslam, Simon A Jones, et al.
Journal of Inherited Metabolic Disease
|
June 22, 2012
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, et al.
Human Mutation
|
July 29, 2017
IDUA mutational profile and genotype-phenotype relationships in UK patients with Mucopolysaccharidosis Type I
Arunabha Ghosh, Jean Mercer, Sabrina Mackinnon, et al.
Journal of Inherited Metabolic Disease
|
January 22, 2011
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H)
Chris J Gardner, Nicola Robinson, Tim Meadows, et al.
Orphanet Journal of Rare Diseases
|
January 22, 2021
Impact of long-term elosulfase alfa treatment on clinical and patient-reported outcomes in patients with mucopolysaccharidosis type IVA: results from a Managed Access Agreement in England
Maureen Cleary, James Davison, Rachel Gould, et al.
Journal of Inherited Metabolic Disease
|
December 21, 2010
Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases
Kia Jane Langford-Smith, Jean Mercer, June Petty, et al.
Molecular Genetics and Metabolism
|
February 3, 2016
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres
Arunabha Ghosh, Weston Miller, Paul J Orchard, et al.
Haematologica
|
February 29, 2012
Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy
Muhammad Ameer Saif, Brian W Bigger, Karen E Brookes, et al.
Orphanet Journal of Rare Diseases
|
April 19, 2015
Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcome
Abhijit Ricky Pal, Eveline J Langereis, Muhammad A Saif, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation
|
February 25, 2015
Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines
Mieke Aldenhoven, Simon A Jones, Denise Bonney, et al.
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of 4