Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trialResearch in context

Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trialResearch in context

Summary: Background: Myotonic dystrophy type 1 (DM1) is a devastating multisystemic disorder caused by a CTG repeat expansion in the DMPK gene, which subsequently triggers toxic RNA expression and dysregulated splicing. In a preclinical study, we demonstrated that erythromycin reduces the toxicity...

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Bibliographic Details
Main Authors: Masayuki Nakamori, Daisaku Nakatani, Tomoharu Sato, Yuhei Hasuike, Seiko Kon, Toshio Saito, Harumasa Nakamura, Masanori P. Takahashi, Eisuke Hida, Hirofumi Komaki, Tsuyoshi Matsumura, Hiroto Takada, Hideki Mochizuki
Format: Article
Language:English
Published: Elsevier 2024-01-01
Series:EClinicalMedicine
Subjects:
Erythromycin
Myotonic dystrophy
RNA toxicity
Splicing
Online Access:http://www.sciencedirect.com/science/article/pii/S2589537023005679

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