A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...

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

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

Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies.

Roseland Community Hospital on Monday celebrated the opening of a new sickle cell treatment clinic. The ribbon was cut at 9 a ...

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

A new study finds that only one in three patients visiting emergency departments (EDs) for severe pain associated with sickle ...

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

The new clinic aims to expand access to specialized care for a community disproportionately affected by sickle cell disease.

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