Tracking a rare disease from birth: study seeks clues for better future care
NCT ID NCT03694561
Summary
This study aims to learn more about the early signs and symptoms of late-onset Pompe disease in babies and young children who are diagnosed through newborn screening. Researchers will closely monitor 20 children over several years to understand how the disease develops and to help determine the best time to start treatments. The study does not provide any treatment itself but gathers information to improve future care guidelines.
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Contacts and locations
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Locations
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Duke University
Durham, North Carolina, 27705, United States
Conditions
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