Risdiplam NEW

As the first small molecule disease-modifying therapy targeting mRNA, Risdiplam operates by binding specifically to two sites on SMN2 precursor mRNA to produce an ample amount of functional protein. Additionally, Risdiplam can penetrate the blood-brain barrier, dispersing into both central and peripheral tissues, systematically increasing the levels of SMN protein throughout the body, which yields more benefits compared to exclusively targeting the central nervous system.