Using iPSC-Derived Cardiomyocytes to Understand DM1

Human iPSC-derived cardiomyocytes recapitulate some features of the DM1 heart.

Regulation of MBNL1 Localization and Function in the CNS

Subcellular compartmentalization of MBNL1 plays a key role in regulation of neurite differentiation and its disruption may contribute to CNS defects in DM.

Tissue-Specific Sensitivities to DM1 Repeat Expansion

New DM1 mouse models demonstrate differential susceptibility of specific cell types to repeat expansion and mis-splicing.

Toward a Cardiac Biomarker for DM1?

Pursuit of a cardiac biomarker for DM may have yielded a biomarker of overall DM1 severity.

DM and the Canadian Neuromuscular Disease Registry

A new publication details organization of and data from a Canadian neuromuscular disease registry.

Predictors of DM1 Patient Survival

A new scoring system identifies key risk factors and a novel tool for assessment of DM1 patient life expectancy.

Speech Disorders in Congenital and Childhood DM1

Speech disorders (dysarthria) in CDM and childhood-onset DM1 have long been recognized and surveillance by speech and language therapists is an important aspect of patient care. Facial weakness and myotonia, and involvement of oral cavity, palatopharyngeal and respiratory muscles, are known to contribute to speech impairment.

Comorbidity of Childhood DM1 and Autism?

After a new review of the literature, the question of comorbidity of childhood DM1 and autism spectrum remains an open one.

Toward ‘Responsive’ Outcome Measures for DM1

Discovery of the holy grail for DM therapy development-- drug registration endpoints -- lies in the dogged pursuit and sharing of natural history data.

GSK3β as a Drug Development Target for DM1

A new study examines the role of the GSK3β—cyclin D3—CUGBP1 pathway in the pathogenesis of DM1 and its potential as a therapeutic target.

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