Plasma and urinary levels of dermatan sulfate and heparan sulfate derived disaccharides after long-term enzyme replacement therapy (ERT) in MPS I: correlation with the timing of ERT and with total urinary excretion of glycosaminoglycans

Hematopoietic stem cell transplantation improves the high incidence of neutralizing allo-antibodies observed in Hurler's syndrome after pharmacological enzyme replacement therapy

(2012) M. A. Saif et al.   HAEMATOLOGICA

Enzyme Replacement Therapy and/or Hematopoietic Stem Cell Transplantation at diagnosis in patients with Mucopolysaccharidosis type I: results of a European consensus procedure

(2011) Minke H de Ru et al.   Orphanet Journal of Rare Diseases

Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases

(2010) Kia Jane Langford-Smith et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Dermatan sulfate and heparan sulfate as a biomarker for mucopolysaccharidosis I

(2010) Shunji Tomatsu et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

(2009) Robert F. Wynn et al.   JOURNAL OF PEDIATRICS

Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

(2009) Kia Langford-Smith et al.   MOLECULAR GENETICS AND METABOLISM

Validation of disaccharide compositions derived from dermatan sulfate and heparan sulfate in mucopolysaccharidoses and mucolipidoses II and III by tandem mass spectrometry

(2009) Shunji Tomatsu et al.   MOLECULAR GENETICS AND METABOLISM

Heparin cofactor II–thrombin complex: A biomarker of MPS disease

(2008) Derrick R. Randall et al.   MOLECULAR GENETICS AND METABOLISM

A dose-optimization trial of laronidase (Aldurazyme®) in patients with mucopolysaccharidosis I

(2008) Roberto Giugliani et al.   MOLECULAR GENETICS AND METABOLISM

Disaccharide structure code for the easy representation of constituent oligosaccharides from glycosaminoglycans

(2008) Roger Lawrence et al.   NATURE METHODS

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

(2008) L. A. Clarke et al.   PEDIATRICS

Mucopolysaccharidosis I: Management and Treatment Guidelines

(2008) J. Muenzer et al.   PEDIATRICS