Towards development of a statistical framework to evaluate myotonic dystrophy type 1 mRNA biomarkers in the context of a clinical trial

Myotonic dystrophy type 1 (DM1) is a rare genetic disorder, characterised by muscular dystrophy, myotonia, and other symptoms. DM1 is caused by the expansion of a CTG repeat in the 3’-untranslated region of DMPK. Longer CTG expansions are associated with greater symptom severity and earlier age at o...

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Detalles Bibliográficos
Autores principales: Kurkiewicz, Adam, Cooper, Anneli, McIlwaine, Emily, Cumming, Sarah A., Adam, Berit, Krahe, Ralf, Puymirat, Jack, Schoser, Benedikt, Timchenko, Lubov, Ashizawa, Tetsuo, Thornton, Charles A., Rogers, Simon, McClure, John D., Monckton, Darren G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7156058/
https://www.ncbi.nlm.nih.gov/pubmed/32287265
http://dx.doi.org/10.1371/journal.pone.0231000

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