People diagnosed with myotonic dystrophy type 1 have difficulty unclenching muscles due to a genetic defect that generates toxic material within their cells. There is currently no treatment. In a new report published in the Proceedings of the National Academy of Sciences on Monday, a group at Scripps Research in Florida reports making a potential drug that targets its key disease-causing RNA. In mouse and cellular models of myotonic dystrophy type 1, it improved the muscle defects with no apparent side-effects.
* This article was originally published here
