Experimental drug offers hope for rare brain development disorder in toddlers
NCT ID NCT02396459
Summary
This study is testing whether a drug called tiratricol can help young boys with a rare genetic disorder called Allan-Herndon-Dudley Syndrome. The condition causes severe brain development problems and harmful overactivity in other body systems. Boys aged 30 months or younger will take the drug for nearly two years to see if it improves their movement skills and brain development while reducing harmful effects on the body.
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Contacts and locations
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Locations
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Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Berlin, 13353, Germany
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Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine
Prague, 15006, Czechia
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Erasmus MC
Rotterdam, 3015 GD, Netherlands
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Oregon Health & Science University (OHSU) Doernbecher Childrens Hospital
Portland, Oregon, 97239, United States
Conditions
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