Discovery of risdiplam (evrysdi) - A medicine for the treatment of spinal muscular atrophy

Tue19  Oct11:00am(30 mins)
Where:
Hall 1B
Dr Hasane Ratni

Abstract

SMA is an inherited disease that leads to loss of motor function and ambulation, and a reduced life expectancy. We have been working to develop orally-administrated, systemically-distributed small molecules to increase levels of functional SMN protein. Herein, we describe the discovery risdiplam that focused on thorough pharmacology, DMPK and safety characterization and optimization. This molecule is the world first approved small molecule RNA splicing modulator.
To view the video assoicated with this lecture click here
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