The NHS has approved a gene therapy called exagamglogene autotemcel, or exa-cel, as a treatment for sickle cell disease. This one-time therapy alters patients' stem cells to address the faulty gene causing the disease. Annually, around 50 patients, primarily older children and adults without matched donors, are expected to benefit. Clinical trials suggest a remarkable 96.6% success rate, stopping painful sickle cell crises. Sickle cell disease presents significant health challenges, largely affecting those of African or Caribbean descent, with 17,000 living with it in England.
A groundbreaking 1.65m treatment offering a potential cure for people in England living with sickle cell disease has been approved for use on the NHS.
The therapy can stop painful sickle cell crises, with researchers finding that there was a functional cure in 96.6% of participants who received the treatment.
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