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The goals of this program are to characterize and describe the genetic disorder mucopolysaccharidosis type IV, A (MPS IVA), also known as Morquio syndrome, to better understand its progression and natural history. For participants taking Vimizim®, an enzyme replacement therapy, the study will also evaluate its long-term effectiveness. People with MPS IVA lack an enzyme known as GALNS that normally breaks down a certain type of sugar, leading to its accumulation in body tissues and causing physical problems such as: decreased growth; eye, throat, and ear problems; heart disease; lung problems; and bone and joint problems. Through this program, patient test results will be entered into a database along with other MPS IVA patients. Analysis of the collected information may lead to insights that will help physicians learn more about treating people with the condition.
Subject to certain exclusion criteria, the study is accepting patients of both genders and of all ages who have a confirmed diagnosis of MPS IVA.
Males and Females: All ages
Physicians must provide medical history information about their enrolled patients. Information from other tests required over time for disease management will be entered into the database. For participants who are also receiving enzyme replacement therapy, additional blood work may optionally be collected for analysis and entered into the study database.
Visits: Not applicable
Duration: 10 years
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.