Sleep studies are indicated for DM1 patients with disrupted sleep or daytime sleepiness in order to direct selection of the optimal treatment regimen.
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.