Highlights•Twenty candidate drugs are in current preclinical and clinical phases in
DM1.•Three new interventional first-in-human clinical trials got underway during 2021 …

Myotonic Dystrophies type 1 (DM1) and type 2 (DM2) are complex multisystem diseases
without disease-based therapies. These disorders are caused by the expansions of unstable …

Muscles undergo developmental transitions in gene expression and alternative splicing that
are necessary to refine sarcomere structure and contractility. CUG-BP and ETR-3-like …

Abstract Myotonic dystrophy type 2 (DM2) is an autosomal-dominant multisystemic disease
with a core manifestation of proximal muscle weakness, muscle atrophy, myotonia, and …

Current studies concerning myotonic dystrophy type 1 (DM1) are in the process of
transitioning from molecular investigations to preclinical and clinical trials. Several papers …

Purpose of review This review aimed to summarize the clinical characteristics of myotonic
dystrophy type 1 and to provide a comprehensive review of the current management options …

The multisystemic aspects of the disease require a multidisciplinary approach for some
patients, most likely even including state-of-the-art cardiac and brain imaging to detect and …

BACKGROUND Myotonic dystrophy type 1 (DM1) is a genetic neuromuscular disease
involving multiple systems, especially the cardiopulmonary system. The clinical phenotype …

Introduction Myotonic dystrophy type 1 (DM1) is the most prevalent muscular dystrophy in
adults. People with DM1 might represent a high-risk population for respiratory infections …

Myotonic dystrophy type 1 (DM1) is an autosomal dominant hereditary disease caused by
abnormal expansion of unstable CTG repeats in the 3′ untranslated region of the myotonic …