RG7916 Targets Underlying Genetic Cause of Type 2 and 3 SMA, Preliminary Trial Results Show
Preliminary results from a phase 2 trial confirm that the investigational drug RG7916 targets the underlying genetic cause of type 2 and type 3 spinal muscular atrophy (SMA), PTC Therapeutics has announced. RG7916 is an oral drug that modulates the splicing of the SMN2 gene to increase production…