With many potential drugs and treatments in development, this session explores the different stages of the clinical trials process, how COVID-19 has impacted the research process as well as the FDA review/approval process, how the different therapeutic approaches might be administered, as well as how people living with myotonic dystrophy can prepare now to be ready to participate in trials in the future.
Presented during the Understanding DM Research and Progress Track at the MDF 2020 Virtual Conference.
About the presenter:
Nicholas Johnson, MD
Dr. Nicholas Johnson treats adults and children with both common and rare neuromuscular conditions, yet his work doesn’t end in the clinic. He dedicates significant time each week to laboratory research, and is part of a team at VCU Health working to advance the treatment of genetic muscle disorders, with a special emphasis on muscular dystrophies. Dr. Johnson is board-certified in neurology, neuromuscular medicine and neuromuscular pathology by the American Academy of Neurology, and serves on its government relations committee.
Among many other DM-related projects and research, Dr. Johnson was the PI of the recent myotonic dystrophy prevalence study and is currently the Co-PI of the END-DM1 Study (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1), a non-interventional natural history study designed and run by the Myotonic Dystrophy Clinical Research Network (DMCRN), a network of medical centers that aims to support future clinical trials of potential therapies for DM1 through the generation of evidence around endpoint measures and testing methods.