[Clinical study of enzyme replacement therapy with idursulfase].

  title={[Clinical study of enzyme replacement therapy with idursulfase].},
  author={Luis Gonz{\'a}lez Guti{\'e}rrez-Solana},
  journal={Revista de neurologia},
  volume={44 Suppl 1},
INTRODUCTION Important advances have been made in enzyme replacement therapy in the treatment of lysosomal diseases over the last two decades. Here we review the initial ERT trial using idursulfase in Hunter syndrome (mucopolysaccharidosis type II) and we also examine relevant aspects of the use of this enzyme. DEVELOPMENT A preclinical trial in a knockout mouse showed a decrease in glycosaminoglycans, both in urine and in tissues, following treatment with idursulfase. In a randomised, double… CONTINUE READING