FDA released an online course to accompany their draft guidance on how to best communicate with the Agency in preparing and filing an IND.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?
MDF's youngest fundraiser? Six-year-old Asher Adleberg! The MDF community has done an amazing job of hosting parties, golf tournaments, crawfish boils, foot races and other events to support Care and a Cure for DM. We have some amazing stories to share, and some fun upcoming events you might want to put on your calendar.
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.
Sleep studies are indicated for DM1 patients with disrupted sleep or daytime sleepiness in order to direct selection of the optimal treatment regimen.