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Gene therapy research grant information

Calliope Joy Foundation Request for Proposal August 2017 Background Leukodystrophies are a group of disorders affecting the brain’s white matter (or myelin). The word comes from "leuko" (Greek for white) and "dystrophy" meaning "imperfect growth." Therefore, leukodystrophies are characterized by imperfect development of the brain's white matter. Children with leukodystrophy appear healthy and achieve their milestones but then lose the ability to crawl, walk, swallow, speak, eat, smile, and laugh. There are more than 30 forms of the disease, and 1 in 7400 live births are affected. In most cases, once children develop symptoms they cannot be treated since the damage to the brain and central

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