Mucopolysaccharidosis VII Disease Monitoring Program
Recruiting
Observational Study
Mucopolysaccharidosis VII
MPS VII
MPS 7
Sly Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 50 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
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 patient…
Contacts