A new clinical trial planning grant initiative from the National Institute of Nursing Research (NINR) provides opportunities to develop an evidentiary basis for the consensus-driven clinical care recommendations currently under development at MDF for DM1.
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.
In 2016, MDF’s Warmline received many phone calls from MDF community members who struggled to complete the application process for federal and state disability benefits, and who needed guidance on how to navigate the complex application process.
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.