Mucopolysaccharidosis I (MPS I) is a rare disease where the body lacks a specific enzyme called alpha-L-iduronidase, which helps break down certain sugars. The MPS I Registry is a global database that collects information about people with this disease. By tracking patient outcomes, the registry helps researchers understand how the disease progresses and how treatments, like enzyme replacement therapy with Aldurazyme, work.
The study does not involve new treatments but gathers data from patients receiving regular care. There are no special visits or extra tests required for joining the registry.
- Participation is open to anyone diagnosed with MPS I, with no exclusion criteria.
- Participants must provide a signed authorization form.
- Being in this registry won't stop you from being in other studies.
For more information, or to join the registry, people can contact their regional representative (e.g., Vivian Liu in Asia-Pacific, or others in Europe, Latin America, and North America) using the provided phone numbers or emails. This registry aims to improve understanding and care for those with MPS I by analyzing long-term data on treatment effectiveness and patient safety.