Previous studies show that [MBNL] inferred can vary over time within individuals with CDM, with an overall trend toward improvement in early childhood, followed by increasing variability beginning around age 7 years. 18,20 To examine the relationship between early childhood motor performance and variability of splicing dysregulation in adolescence, analyses involving [MBNL] inferred were completed in participants 6 years and older.
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Toward Trial Readiness in Congenital Myotonic Dystrophy: A Longitudinal Cohort Study of Predictors of Motor Function in Childhood.
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