Individualization of long-term enzyme replacement therapy for Gaucher disease

  title={Individualization of long-term enzyme replacement therapy for Gaucher disease},
  author={Hans Christer Andersson and Joel Charrow and Paige Kaplan and Pramod Mistry and Gregory M. Pastores and Ainu Prakesh-Cheng and Barry E Rosenbloom and C. Ronald Scott and Rebecca S. Wappner and Neal J Weinreb},
  journal={Genetics in Medicine},
Gaucher disease, the most common lysosomal storage disorder, is a heterogeneous condition affecting multiple organ systems. Patients with nonneuronopathic (type 1) Gaucher disease may suffer from hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Enzyme replacement therapy (ERT) with mannose-terminated glucocerebrosidase (imiglucerase, Cerezyme, Genzyme Corporation… CONTINUE READING