Molecular Genetics Model of CDM

A review synthesizes the literature into a molecular genetics model of CDM and suggests more answers lie in research on DMPK locus epigenetics.

Status Report: Genome Editing in DM1

A concise summary of genome editing efforts in DM1 to date and a look at gaps in getting to an approved therapeutic.

How to Talk about a DM1 Diagnosis

A new study provides insights into how families communicate a diagnosis of DM1.

Upstream Methylation Impacts Muscle and Respiratory Function in DM1

Epigenetic modifications linked to the pathomechanism of CDM may also play a role in the severity of DM1.

Sizing Expanded DMPK Repeats

Data validating a novel commercial method for sizing expanded DMPK repeats in DM1 is published.

The DM-Scope Registry Model

The DM-Scope registry model supports the leveraging of clinical research to improve both therapeutic development and patient care.

A Genetic Modifier of DM1

A MSH3 variant modifies expanded repeat stability and age of disease onset in HD and DM1.

A Tighter Relationship Between CTG Repeat Length and DM1 Phenotype?

Occurrence of interruptions in expanded CTG repeat tracts represents a key factor in understanding genotype/phenotype correlations in DM1.

MDF Publishes First-ever CDM and DM2 Clinical Care Recommendations

MDF is pleased to announce the publication of the first-ever Consensus-based Care Recommendations for Congenital and Childhood-onset Myotonic Dystrophy Type 1 and Myotonic Dystrophy Type 2.

Linking DM Molecular Events to Insulin Resistance and Muscle Atrophy

Defective insulin signaling activation may underlie skeletal muscle wasting in DM1 and DM2.

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