Last updated on December 2018

Mucopolysaccharidosis I (MPS I) Registry


Brief description of study

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.

The objectives of the Registry are:

  • To evaluate the long-term effectiveness and safety of Aldurazyme (laronidase)
  • To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I
  • To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

Detailed Study Description

The MPS I Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

Clinical Study Identifier: NCT00144794

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Investigational Site Number 152001
Talcahuano, Chile
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Recruitment Status: Open


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