Offered by: Nemours Children's
Location: Delaware Valley
Morquio A Registry Study (MARS)
The goal of the registry is to collect medical information on people with Morquio syndrome from around the world in an effort to better understand what happens to them over time and to help their doctors take care of them.
Any individual diagnosed with Morquio A syndrome (MPS IVA) can be in the study.
The registry will last about 10 years. Registry information and data collection will start at enrollment in the registry. Some of the data for the baseline assessments may be collected from data obtained prior to registry enrollment. Data for the registry visits will be collected every 6 months.
If you decide to join the registry, information and results from the tests listed below and any additional tests that your doctor may recommend to monitor your health will be given to the sponsor and entered in the registry database. Not all patients will have all tests described. Your doctor will decide which tests you need at each follow up visit.
The following assessments may be conducted during the registry: physical examination, anthropometric measurements, current medications, vital signs, medical history, health questionnaires, mechanical ventilation use, information on receiving enzyme replacement therapy, blood and urine tests, endurance tests, eye exams, hearing tests, heart exams, dental exams, respiratory exams, sleep tests, and imaging such as MRI, CT, ultrasound or X-rays.
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