Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

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

Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...

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

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

Objective: To review the pathophysiology of anemia, as well as transfusion-related complications and indications for red blood cell (RBC) transfusion, in critically ill children. Although allogeneic ...

The patient died from vent-occlusive liver disease, a well-characterized side effect of the busulfan, the myeloablative chemotherapy used to prepare the bone marrow for engraftment with the ...

Starting iron chelation therapy early may prevent growth problems and organ damage from iron overload in children with TDT. Currently approved medications have limitations for use in very young ...

A senior doctor says 14 children who received blood transfusions at hospitals in northern India contracted hepatitis and HIV infections. The children, aged between six and 16 years, were all being ...