Mucopolysaccharidosis Type VII Disease Monitoring Program (MPS VII DMP)

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Mucopolysaccharidosis Type Vii (mps Vii)
  • Age: Between 0 - 100 Years
  • Gender: Male or Female
  • Other Inclusion Criteria:
      Confirmation of MPS VII disease based on glucuronidase enzyme assay (e.g., leukocyte or fibroblast), and if not confirmed, then mutation testing. Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or 16 years, depending on the region) or patients >18 years of age who have cognitive deficiencies, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures. Willing and able to comply with study visit schedule Willing to comply with study procedures depending on developmental and physical capability

You may not be eligible for this study if the following are true:

    • Concurrent enrollment in an Ultragenyx-sponsored clinical trial Participation in any other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx Any comorbid disease or condition that, in the view of the physician, would interfere with study participation or would affect safety of the patient.



If you are registered as a volunteer, please login to the dashboard to send referrals.

Contact the research team to learn more about this study.

By clicking "Contact Research Team", your contact information will be sent securely to the research staff associated with the study. You will also receive a copy of this email in your inbox, as well as other notifications to determine your participation status in the study.