Condition: Mucopolysaccharidosis IV Type A · Morquio A Syndrome · MPS IVA · Sponsor: Nemours Children's Clinic
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, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal/pulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.
This description comes directly from the study's public registry record.
Shunji Tomatsu, MD PhD · 3022987336 · stomatsu@nemours.org
Greg Stets · 302-298-6504 · gstets@nemours.org
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| Nemours Children's Health, Delaware Valley | Wilmington, Delaware, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05284006