Givosiran was approved by the US FDA for the treatment of acute hepatic porphyria, a rare generic blood disorder, nearly three months ahead of the action date.

A series of basic research insights around the cell death process called ferroptosis could form the underpinnings of a new class of treatments aimed at drug resistant tumors.

Recent clinical data using antisense drugs in neurological diseases are encouraging signs that targeting genetic drivers of ALS with antisense oligonucleotides or other gene-altering drugs could halt progression of several forms of the disease.