Chimeric proteins elucidate mechanisms of MBNL binding and regulation of alternative splicing, and suggest a path to optimize therapeutic strategies for DM.
Conceptually sound strategies exist to repair or replace defective genes that cause disease—what will it take to ensure these are safe and effective?
A novel optical imaging approach helps characterize the central nervous system phenotype of DM2 mice.
MDF holds an offsite planning meeting in January every year to look at the Care and Cure landscape for myotonic dystrophy. The annual goals are to identify urgent and high-impact opportunities to improve the quality of life of every person living with this disease while continuing to accelerate the search for therapies.
After a new review of the literature, the question of comorbidity of childhood DM1 and autism spectrum remains an open one.