A review synthesizes the literature into a molecular genetics model of CDM and suggests more answers lie in research on DMPK locus epigenetics.
A concise summary of genome editing efforts in DM1 to date and a look at gaps in getting to an approved therapeutic.
A new study provides insights into how families communicate a diagnosis of DM1.
Epigenetic modifications linked to the pathomechanism of CDM may also play a role in the severity of DM1.
Data validating a novel commercial method for sizing expanded DMPK repeats in DM1 is published.
The DM-Scope registry model supports the leveraging of clinical research to improve both therapeutic development and patient care.
A MSH3 variant modifies expanded repeat stability and age of disease onset in HD and DM1.
Occurrence of interruptions in expanded CTG repeat tracts represents a key factor in understanding genotype/phenotype correlations in DM1.
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.
Defective insulin signaling activation may underlie skeletal muscle wasting in DM1 and DM2.