Next Generation Splicing Modulators
Time: 11:00 am
day: Day Two
Details:
- The approval of Risdiplam for the treatment of SMA validated the approach of selectively modulating splicing with small molecules
- PTC has developed a comprehensive discovery platform to identify novel splicing modulators for the treatment of diseases with unmet needs
- Novel splicing modulators are being optimized for recently identified disease-relevant splicing events
