It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient's body.
Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it down for ...
“The war we’re in right now is much harder,” said Arab, who heads the Iranian Thalassaemia Society, which represent those with the blood disorder. “Innocent people are dying, not because ...
when a haematopoietic stem cell transplant (a blood and marrow transplant) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available. More evidence on ...
Next review: More evidence on exagamglogene autotemcel is being collected. After this, NICE will decide whether to recommend it for use in the NHS and update the guidance. It will be available with ...