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Cognitive and Neurological Pathologies in Pompe Disease
Sponsor: Duke University
Summary
The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. The investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.
Key Details
Gender
All
Age Range
0 Years - 18 Years
Study Type
OBSERVATIONAL
Enrollment
45
Start Date
2016-08-03
Completion Date
2029-03
Last Updated
2026-03-04
Healthy Volunteers
No
Conditions
Locations (1)
Duke University Medical Center
Durham, North Carolina, United States