Dutch biotech Pharming has claimed FDA approval for Joenja, the first drug indicated for recently identified rare immunodeficiency disorder activated phosphoinositide 3-ki
The US FDA announced today that it has cleared two novel gene therapies for sickle cell disease: Vertex Pharmaceuticals' Casgevy and bluebird bio's Lyfgenia.
On 1st October, the roll-out of the National Contract Value Review (NCVR) process marked a pivotal moment in the trajectory of clinical research in the UK, introducing a standardised, natio