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Mucopolysaccharidosis VII Disease Monitoring Program

Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Who can join

All ages · All sexes

Inclusion Criteria:

* Diagnosis of MPS VII based on laboratory diagnosis, including either enzymatic or mutation analysis.
* Willing and able to provide written informed consent or, in the case of patients under the age of 18 (or below adult ages as defined by local laws and regulations) 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 DMP has been explained, and prior to any research-related procedures.
* Willing to comply with DMP visit schedule.

Exclusion Criteria:

* Concurrent participation in other pharmaceutical company-sponsored interventional clinical trial unless approved by Ultragenyx.

About the study

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

What is being tested

• No Intervention (other)

Sponsor: Ultragenyx Pharmaceutical Inc · Participants: 50 · Started: Jan 29, 2018

Contact the study team

• Patients Contact: Trial Recruitment · Phone: 1-888-756-8657
• HCPs Contact: Medical Information · Phone: 1-888-756-8657

Official record on ClinicalTrials.gov — NCT03604835

Locations in the U.S.

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.