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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.

Determinants of Respiratory Function in DM1

Close monitoring of respiratory function reduces respiratory complications in DM1.

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.

Martha Montag Brown: A Recruiter Gets Recruited

As Matthew Brown listened to his cousin Emily describe the symptoms that led to doctors diagnosing her with having multiple sclerosis, he grew concerned. The problems she described—an inability to relax her hand, a freezing of her tongue that made it impossible to talk at times, and gastrointestinal issues—were the same ones he had been experiencing.

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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