Scientists create potent molecules aimed at treating muscular dystrophy | 21st Century Innovative Technologies and Developments as also discoveries, curiosity ( insolite)... |
While RNA is an appealing drug target, small molecules that can actually affect its function have rarely been found.


But now scientists from the Florida campus of The Scripps Research Institute have for the first time designed a series of small molecules that act against an RNA defect directly responsible for the most common form of adult-onset muscular dystrophy.