Cognitive and Neurological Pathologies in Pompe Disease
Recruiting
Observational Study
Pompe Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 0 – 18
- Sex
- Any
- Study type
- Observational
- Participants needed
- 45 (estimated)
- Sponsor
- Duke University · Other
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About This Trial
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 …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)
Exclusion Criteria:
* Refusal to give informed consent
Contacts