Bone manifestations in neuronopathic Gaucher disease while receiving high-dose enzyme replacement therapy

Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's Disease

Norman W. Barton, Roscoe O. Brady, James M. Dambrosia et al. · New England Journal of Medicine · 1991 · 1.3K citations · Full text

Life Sciences, Biochemistry, Pharmacotherapy +17

Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

Neal J. Weinreb, Joel Charrow, Hans C. Andersson et al. · The American Journal of Medicine · 2002 · 541 citations

Life Sciences, Genetics, Pharmacotherapy +9

Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1

Hans C. Andersson, Paige Kaplan, Katherine Kacena et al. · PEDIATRICS · 2008 · 166 citations

Medicine, Hematology, Pediatrics +17

Phenotypic continuum in neuronopathic gaucher disease: an intermediate phenotype between type 2 and type 3

Özlem Göker-Alpan, Raphael Schiffmann, Joseph K. Park et al. · The Journal of Pediatrics · 2003 · 164 citations

Life Sciences, Genetics, Neurology +11

Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry

Anna Tylki‐Szymańska, Ashok Vellodi, Amal El‐Beshlawy et al. · Journal of Inherited Metabolic Disease · 2010 · 144 citations

Life Sciences, Neurology, Neuroscience +9