Casgevy safely and effectively prevents vaso-occlusive crises in children ages 5 to 11 with severe sickle cell disease, trial ...

In October 2024, Mercy’s newborn son was diagnosed with sickle cell disease, an inherited blood disorder caused by a mutation ...

Cambridge-based Fulcrum Therapeutics said it will aim to launch a large clinical trial in the second half of 2026 to prove ...

Data presented at ASH suggest that the CRISPR therapy could provide a one-time functional cure in children as young as 5 ...

Vertex Pharmaceuticals presents clinical data demonstrating benefits of Casgevy in SCD and TDT patients aged 5+ at ASH ...

Less than a third of ED visits for acute sickle cell disease pain were guideline-adherent for the timely administration of ...

Patients who underwent hematopoietic cell transplantation for sickle cell disease saw high rates of survival without disease ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Exagamglogene autotemcel helped children with beta thalassemia achieve transfusion independence and prevented vaso-occlusive crises among those with sickle cell disease in a pair of phase 3 ...

Most people with sickle cell disease who undergo allogeneic hematopoietic cell transplantation achieve favorable long-term ...

Casgevy, now a "national priority" drug, helped kids with sickle cell and thalassemia, results which may help toward a label ...