Non-invasive Functional Assessment and Pathogenesis of Morquio A
Recruiting
Observational Study
Mucopolysaccharidosis IV Type A
Morquio A Syndrome
MPS IVA
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
- 60 (estimated)
- Sponsor
- Nemours Children's Clinic · Other
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About This Trial
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperl…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patients affected by MPS IVA. The diagnosis of MPS IVA is confirmed by deficient enzyme activity of \< 5% of normal activity level as measured in plasma or leukocytes.
Exclusion Criteria:
* No
Contacts