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 ...

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 ...

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 ...