Hundreds of sufferers of a severe blood disorder given hopes of a cure as 'life-changing' gene therapy is set to be made available on the NHS By Kate Pickles Health Editor For The Daily Mail Published: 00:29, 8 August 2024 | Updated: 00:30, 8 August 2024 e-mail View comments Hundreds of sufferers of a severe blood disorder have been give hopes of a cure as ‘life-changing’ gene therapy is set to be made available on the NHS . Experts say Casgevy - which costs £1.65million per treatment - fixes a faulty gene in the patient’s bone marrow stem cells, stopping the need for regular transfusions and improving life expectancy.
It has been approved for use in patients with severe beta-thalassaemia when no bone marrow transplant is available. Health leaders said it was a ‘historic moment’ for those with the life-limiting condition, with the UK becoming the first country in Europe to offer the treatment. Transfusion-dependent beta thalassemia (TDT) is a severe inherited blood disorder that requires lifelong red blood cell transfusions to survive.
It is caused by mutations in the beta-globin gene, which affects the body’s production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body. Sufferers of a severe blood disorder have been given hope of a cure as 'life-changing' gene therapy has been made available on the NHS (file photo) Exagamglogene autotemcel, or exa-cel as it is otherwise known, has been recommended by Nice to treat patients aged.