Blood disorders affect millions of Americans, yet many don’t realize these conditions can sometimes transform into something far more serious. With approximately 1.3 million people currently living ...

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

The National Institute for Health and Care Excellence (NICE) has recommended exagamglogene autotemcel (Casgevy, Vertex) – commonly known as 'exa-cel' – to treat transfusion-dependent beta thalassaemia ...

A Roche drug in development for treating a rare blood disorder has Phase 3 data showing it worked at least as well as a blockbuster AstraZeneca drug while also offering patients a dosing advantage.

This month the FDA has approved a treatment for an inherited blood disorder. The Casgevy treatment was developed through CRISPR gene-editing technology and involves modifying a person’s own blood stem ...