Clinical trials involve substantial investments by drug developers, but especially by DM patients and families. Clinical trials proposed for our community must have adequate scientific rationale behind them.
MDF runs down some important changes in the purposes and rationale of NINDS funding mechanisms for postdoctoral fellows.
FDA released an online course to accompany their draft guidance on how to best communicate with the Agency in preparing and filing an IND.
Gene editing is a potential avenue for therapy development in DM. With the safety, efficacy and delivery challenges, how do we get there?
When Dr. Thurman Wheeler was a resident in neurology, he remembers a senior physician telling him that myotonic dystrophy would probably be one of the most difficult diseases to treat because it involves so many body systems.