Cognitive and Adaptive Development in Congenital/Childhood DM1

A new longitudinal study focuses on CNS function in congenital and childhood DM1.

A Novel Genome Editing Strategy for DM1

A novel genome editing strategy removes expanded CUG repeats in DMPK transcripts.

Corticospinal Tract Involvement in DM1

DTI detects specific changes in the corticospinal tract, reflecting cortical gray matter volume reductions linked to DM1 motor function deficits.

Challenges in Assessing the CNS in DM1

Development, validation, and use of CNS clinical outcome assessments in DM1 must consider their interactions with other parameters of patient functioning.

What We Do (and Don’t) Know About Cognitive Function in DM1

A literature review suggests meta-analysis of cognitive studies in DM1 is both feasible and can inform understanding of disease mechanisms and patient management.

Non-Invasive Biomarkers for DM1

A new study lends support for a DM biomarker panel based upon patient urine exRNA assays that may serve as a pharmacodynamic biomarker for go/no go decisions in drug development for DM1.

Review: What Have We Learned About RAN Translation?

A new review looks at the cross-disease evidence for RAN translation’s contributions to pathogenesis.

MDF Releases DM1 Care Recommendations

MDF is pleased to announce the publication of the first-ever Consensus-based Care Recommendations for Adults with Myotonic Dystrophy Type 1.

Furamidine Mechanism of Action in DM1

A new publication finds a complex mechanism of action of furamidine in DM1 models.

Determinants of Respiratory Function in DM1

Close monitoring of respiratory function reduces respiratory complications in DM1.

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