Studies of AMPK/mTORC1 signaling in DM1 identify novel therapeutic targets for DM, and may offer an opportunity to repurpose approved drugs for both muscle and cognitive symptoms.
2016 was a banner year for the Myotonic Dystrophy Foundation. We completed our 10th year driving Care and a Cure for people and families living with DM, wrapped up year 2 of a 3-year, $5M drug development acceleration effort and oversaw the largest expansion of our Care programming and resources ever.
Biomarkers of various Contexts of Use are essential for drug development in DM—recent guidance documents and publications point to exciting new opportunities.
Longitudinal assessment of cognitive function in adult- and late-onset DM1 reveals a pattern of cognitive decline that can be modeled as an early-onset and acceleration of normal aging.
Scientists from a consulting firm with considerable experience in evaluation of patient-reported outcome measures evaluated available PROMs for their potential in DM1 clinical trials.