Nicholas Johnson, MD, Associate Professor in the Department of Neurology at Virginia Commonwealth University, explains myotonic dystrophy type 1 and how it is currently managed.
DM1 is a progressive neuromuscular disease caused by a triple repeat in the DMPK gene. Symptoms and severity are highly variable among patients. However, all forms of DM1 are associated with high levels of disease burden and may lead to premature mortality. Common symptoms include muscle weakness, cardiac conduction abnormalities, myotonia, cataracts, respiratory problems, fatigue, hypersomnia, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people with DM1.
Data from the phase 1/2 MARINA trial testing AOC 1001 in patients with DM1 was recently presented at the 75th American Academy of Neurology (AAN) Annual Meeting and showed the drug to be safe and effective. A Phase 3 clinical trial is currently being planned.
To stay up-to-date on the latest information about DM1 and other musculoskeletal conditions, go to checkrare.com/diseases/musculoskeletal-diseases/
