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 TERMINÉ 
Leukodystrophies (LDs) are inherited rare neurodegenerative diseases of the white matter and its main component, the myelin, that are affecting predominantly children. Severity of the disease is related to the axonal dysfunction due to myelin deficiency or destruction. Despite the achievement of remarkable advances made in the past decade, there is no current curative therapy. The development of t ...
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 TERMINÉ 
"Only for Children Pharmaceuticals, a French based Small and Medium-sized Enterprise (SME) developing drug for children, is the Coordinator of a multinational, multidisciplinary and complementary Consortium. This Consortium is composed of highly qualified organizations and notorious clinicians in pediatric hemato-oncology. The Consortium is an integrated team of competencies (clinicians, pædiatric ...
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