A potentially revolutionary technology may allow development of a drug for DM that can correct a patient’s DNA by selectively removing the expanded CTG and CCTG repeats in DM1 and DM2, respectively.
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.
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.
In partnership with MDF, the Wyck Foundation awarded a number of new DM research grants.