Enzyme-replacement therapy for metabolic storage disorders.

  title={Enzyme-replacement therapy for metabolic storage disorders.},
  author={Roscoe O. Brady and Raphael Schiffmann},
  journal={The Lancet. Neurology},
  volume={3 12},
BACKGROUND After many years of intensive investigation, enzyme-replacement therapy (ERT) has become standard treatment for patients with type 1 (non-neuronopathic) Gaucher's disease. ERT has greatly changed the clinical course of this disorder by reducing hepatosplenomegaly, by improving anaemia and thrombocytopenia, and by ameliorating skeletal damage. This example has prompted the investigation of ERT for several other metabolic disorders. The results of several of these trials have recently… CONTINUE READING


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