Simon Jones

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

Mieke Aldenhoven, Robert Wynn, Paul J. Orchard et al. · Blood · 2015 · 317 citations · Full text

Medicine, Hematology, Stem Cell Transplantation +15

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

Minke H. de Ru, Jaap Jan Boelens, Anibh M. Das et al. · Orphanet Journal of Rare Diseases · 2011 · 234 citations · Full text

Medicine, Hematology, Pathology +8

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease

Maurizio Scarpa, Zsuzsanna Almássy, Michael Beck et al. · Orphanet Journal of Rare Diseases · 2011 · 231 citations · Full text

Life Sciences, Neurology, Pathology +17

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

Yoav H. Messinger, Nancy J. Mendelsohn, William J. Rhead et al. · Genetics in Medicine · 2012 · 211 citations · Full text

Life Sciences, Immunology, Pediatrics +12

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo‐controlled study

Christian J. Hendriksz, Barbara K. Burton, Thomas R. Fleming et al. · Journal of Inherited Metabolic Disease · 2014 · 211 citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +12