Not too long ago, the concept of selectively targeting mRNA with small molecules 
was perceived as a formidable scientific challenge. The discovery of small 
molecule splicing modifiers and the development of risdiplam for the treatment 
of spinal muscular atrophy (SMA) have firmly established proof of concept for 
this exciting new platform and transformed a scientific curiosity into a viable 
technology to target disease. Today, several approaches to target mRNA with 
small molecules, supported by biophysical and screening methods, are in place to 
deliver new drugs with high therapeutic relevance.