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.
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FDA released an online course to accompany their draft guidance on how to best communicate with the Agency in preparing and filing an IND.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?