Early Discovery Lessons Learned Along the Path to Risdiplam

Time: 9:30 am
day: Day 1 AM


  • With the discovery and commercial success of Evrysdi® (risdiplam) in treating SZA, the use of small molecules to influence pre-mRNA splicing has become a fertile ground for drug discovery
  • With hindsight vision, some of the key determinants to successful identification of risdiplam precursors will be discussed, including the importance of a sensitive screening assay and splicing selectivity
  • Discussing key SAR breakthroughs