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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.

A Tool for Evaluating Drugs Targeting MBNL

Development of a mini gene tool facilitates the identification of candidate therapeutics targeted at dissociating MBNL from expanded CUG repeats.

NIH Releases 2018 Funding Opportunities

NIH has reissued the CREATE Bio, Blueprint Neurotherapeutics and NeuroNEXT initiatives, along with other initiatives that focus on biomarker development and clinical trial readiness, to support therapy development efforts.

Know Your DM1 Repeat Length: It’s Important for Your Cardiac Care

Understanding cardiac and other DM risk factors and planning for the known complications of DM that may affect you can help protect and maintain your quality of life and that of your loved ones.

DM1 Genotype and Cardiac Phenotype

A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.

Do We Treat DM as a Brain Disease?

A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.

The Path We’re On: Many Challenges, but Ultimately a Drug

Taking as many shots on goal as possible is the only path to approval and reimbursement of safe and effective drugs for DM.

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