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

Risk Factor Analysis of Outcomes after Unrelated Cord Blood Transplantation in Patients with Hurler Syndrome

(2009) Jaap Jan Boelens et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Mortality and cause of death in mucopolysaccharidosis type II—a historical review based on data from the Hunter Outcome Survey (HOS)

(2009) S. A. Jones 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

Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice

(2009) Marcelina Malinowska et al.   MOLECULAR GENETICS AND METABOLISM

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

Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II

(2008) J Edmond Wraith   ACTA PAEDIATRICA

Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease

(2008) Ulrich Matzner et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

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

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

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

(2008) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM