FDA adcomm backs accelerated approval of Sareptas Duchenne gene therapy in close vote Endpoints News

X4 Pharmaceuticals announced Monday morning that the FDA approved its daily pill for WHIM syndrome — an ultra-rare immune disease named after its symptoms. The pill, mavorixafor, was approved for…

The FDA approved a Houston drugmaker’s tablet for adults with major depressive disorder last week, capping a decades-long back-and-forth between multiple companies and the regulator over questions of efficacy. On…

The next wave of gene editing technology could come from bacteria living in Antarctic lakes, coal mines or even dog saliva, and those are just a few of the odd…