Researchers at Nemours/Alfred I. duPont Hospital for Children (AIDHC) in Delaware have received funding from the NIH to complete a study to evaluate the natural history of Morquio A syndrome (MPS IVA).
Study aims and objectives are as follows:
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.
Three nights of lodging and travel costs for two people (patient and caregiver) are included, along with the costs of all tests, with participants receiving results from assessments completed.
Contact the study coordinator with any questions: Somto.Jigide@nemours.org