Ashok Vellodi

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Joseph Muenzer, J. E. Wraith, Michael Beck et al. · Genetics in Medicine · 2006 · 585 citations · Full text

Life Sciences, Biochemistry, Enzymology +10

Human Osteoclast-Poor Osteopetrosis with Hypogammaglobulinemia due to TNFRSF11A (RANK) Mutations

Matteo M. Guerrini, Cristina Sobacchi, Barbara Cassani et al. · The American Journal of Human Genetics · 2008 · 307 citations · Full text

Medicine, Molecular Genetics, Molecular Medicine +11

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

Randomized, controlled trial of miglustat in Gaucher's disease type 3

Raphael Schiffmann, Edmond J. FitzGibbon, Chris Harris et al. · Annals of Neurology · 2008 · 233 citations · Full text

Medicine, Neurology, Pharmacotherapy +14

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

Joseph Muenzer, Michael Beck, Christine M. Eng et al. · Genetics in Medicine · 2010 · 232 citations · Full text

Life Sciences, Biochemistry, Enzymology +1