RecruitingNCT04639336

Cognitive and Neurological Pathologies in Pompe Disease


Sponsor

Duke University

Enrollment

45 participants

Start Date

Aug 3, 2016

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Min Age: 0 YearsMax Age: 18 Years

Inclusion Criteria1

  • Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)

Exclusion Criteria1

  • Refusal to give informed consent

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Locations(1)

Duke University Medical Center

Durham, North Carolina, United States

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NCT04639336


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