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.
MDF is excited to participate in Rare Disease Day 2017, an annual event that takes place on the last day of February to raise awareness of rare diseases among policy makers, the scientific community and the general public.
In partnership with MDF, the Wyck Foundation awarded a number of new DM research grants.
Biomarkers of various Contexts of Use are essential for drug development in DM—recent guidance documents and publications point to exciting new opportunities.