A large retrospective study helps clarify genotype-cardiac phenotype relationships in adult DM1.
Susannah is a 48-year-old entrepreneur with her own real estate law practice. She also has two teenagers that keep her busy at home, and she helps care for her mother. Susannah also has DM1.
A recent review article makes the case that DM is a brain disease and that better understanding of and treatment strategies for the neurological consequences of DM are essential.
MDF strives to help National Institutes of Health (NIH) grant applicants compete for research funding.
Taking as many shots on goal as possible is the only path to approval and reimbursement of safe and effective drugs for DM.
MDF staff recently attended the 2017 annual meeting of the American Academy of Neurology, in Boston, MA. Here are highlights from that meeting.
MDF held the first myotonic dystrophy (DM) Patient-Focused Drug Development (PFDD) meeting with key senior leaders from the Food and Drug Administration (FDA) as part of the 2016 MDF Annual Conference in September 2016.