Toward Trial Readiness in Congenital Myotonic Dystrophy: A Longitudinal Cohort Study of Predictors of Motor Function in Childhood
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Background and objectives: Congenital myotonic dystrophy (CDM) is a life-limiting genetic disorder present at birth, marked by profound motor and cognitive impairments. CDM is the most severe form of myotonic dystrophy type 1 (DM... ...
