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.
There have been new discoveries in the way that congenital myotonic dystrophy (CDM) is inherited.
MDF is also happy to help you understand whether something is in a legitimate clinical trial, an approved therapy…or not.
Important review articles on disease mechanisms of and therapy development for DM have recently been published.
The UK DM Patient Registry reports on disease burden for 556 patients with a confirmed diagnosis of DM1.