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Mucopolysaccharidosis I (MPS I) Registry

key information

study id #: NCT00144794

condition: Mucopolysaccharidosis I (MPS I)

status: Recruiting

purpose:

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

results: https://clinicaltrials.gov/ct2/show/results/NCT00144794

last updated: February 26, 2022

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