Scientists track Ultra-Rare, often fatal childhood disease to pave way for future cures
NCT ID NCT03478839
Summary
This study aimed to learn more about two very rare and serious genetic diseases, GACI and ARHR2, which can be fatal in infancy or cause lifelong health problems. Researchers collected medical information from 48 affected individuals and their family members to map out how these diseases progress over time. The goal was to gather essential knowledge to help design and test future treatments for these conditions.
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Contacts and locations
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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