January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...
Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has approved JOURNAVXtm (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal ...
The company’s pipeline includes VX-522, a CFTR mRNA therapeutic designed to treat the underlying cause of CF ... disease and transfusion-dependent beta thalassemia which is in Phase 2/3 clinical ...
When is the Elden Ring Nightreign beta? The Lands Between beckons us once more for a standalone co-op adventure that introduces a roguelike loop into FromSoftware’s formula. Playtests are the ...