Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

  • Protocol
  • Intervention



This is the protocol for a review and there is no abstract. The objectives are as follows:

The objectives of this review will be to assess the effectiveness and safety of ERT with laronidase in the management of patients with:

  1. MPS I who do not undergo HSCT;

  2. MPS I who receive ERT prior to HSCT.