Discovery of the first molecular mechanism of action for a small molecule that improves RNA splicing in cells could lead to new therapeutics for the genetic disorder spinal muscular atrophy (SMA) and ...

Splicing of SMN2 generally skips exon 7, resulting in the production of a truncated and inactivated form of the SMN protein. NVS-SM1, a new compound reported by Palacino and colleagues, facilitates ...

Scientific insights often reflect a shift in perspective. For Envisagenics and its partners, insights may be gained through a more encompassing view of gene expression errors. This view includes ...