RNA mis‐splicing in children with congenital myotonic dystrophy is associated with physical function

RNA mis‐splicing in children with congenital myotonic dystrophy is associated with physical function

Abstract Objectives Dysregulated RNA alternative splicing is the hallmark of myotonic dystrophy type 1 (DM1). However, the association between RNA mis‐splicing and physical function in children with the most severe form of disease, congenital myotonic dystrophy (CDM), is unknown. Methods Eighty‐two...

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Main Authors: Julia M. Hartman, Kobe Ikegami, Marina Provenzano, Kameron Bates, Amanda Butler, Aileen S. Jones, Kiera N. Berggren, Jeanne Dekdebrun, Marnee J. McKay, Jennifer N. Baldwin, Kayla M. D. Cornett, Joshua Burns, Michael Kiefer, Nicholas E. Johnson, Melissa A. Hale, the DMCRN Consortium
Format: Article
Language:English
Published: Wiley 2024-12-01
Series:Annals of Clinical and Translational Neurology
Online Access:https://doi.org/10.1002/acn3.52224
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https://doi.org/10.1002/acn3.52224

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