ArticleNeurology. Genetics2026
Toward Trial Readiness in Congenital Myotonic Dystrophy: A Longitudinal Cohort Study of Predictors of Motor Function in Childhood.
Article in Neurology. Genetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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12 authors.
Funding
Abstract
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 (DM1), both caused by a cytosine-thymine-guanine (CTG) repeat expansion in the DM1 protein kinase gene. Clinical trials targeting the shared disease mechanism in DM1 adults have shown promise. However, a lack of validated clinical end points in early childhood and a limited understanding of the variable disease progression are challenges to trial design in CDM. This longitudinal cohort study identifies predictors of motor function in children with CDM to inform future clinical trials. Methods: Children with genetically confirmed CDM participated in a longitudinal natural history study, completing annual motor assessments, including the 10-m walk/run (10MWRT), 6-minute walk test, supine to stand, and grip strength. Perinatal clinical features and early childhood motor milestone achievement were captured through caregiver proxy report. Linear mixed-effects models were used to evaluate predictors of concurrent motor performance and 1-year change. The relationship between [MBNL] Results: A total of 138 visits from 42 children with CDM aged 3.0-15.3 years were included in the analysis. Motor performance on all motor outcomes improved with age. In age-adjusted models, age at independent walking explained more variance in 10MWRT times ( Discussion: The severity of clinical course in infancy and age of independent walking are key predictors of motor function across childhood in CDM. However, baseline motor performance is the strongest predictor of 1-year change and should be a central consideration in clinical trial design. Larger, prospective studies are needed to assess the responsiveness of early motor milestones as indicators of treatment response.
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