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Clinical improvement of DM1 patients reflected by reversal of disease-induced gene expression in blood

BACKGROUND: Myotonic dystrophy type 1 (DM1) is an incurable multisystem disease caused by a CTG-repeat expansion in the DM1 protein kinase (DMPK) gene. The OPTIMISTIC clinical trial demonstrated positive and heterogenous effects of cognitive behavioral therapy (CBT) on the capacity for activity and...

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Detalles Bibliográficos
Autores principales: van Cruchten, Remco T. P., van As, Daniël, Glennon, Jeffrey C., van Engelen, Baziel G. M., ‘t Hoen, Peter A. C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9646470/
https://www.ncbi.nlm.nih.gov/pubmed/36352383
http://dx.doi.org/10.1186/s12916-022-02591-y