RNA mis-splicing in children with myotonic dystrophy is associated with physical function.
Julia M HartmanKobe IkegamiMarina ProvenzanoKameron BatesAmanda ButlerAileen S JonesKiera N BerggrenJeanne DekdebrunMarnee J McKayJennifer N BaldwinKayla M D CornettJoshua BurnsMichael KieferNicholas E JohnsonMelissa A HalePublished in: bioRxiv : the preprint server for biology (2024)
in DM1. The strength of these correlations and the development of the predictive models will assist in designing efficacious clinical trials for individuals with DM1, particularly CDM.