BSH statement on the MHRA approval of the use of gene editing therapy in sickle cell disease and transfusion-dependent thalassaemia
There is a paucity of available effective treatments for sickle cell disease and thalassaemia both of which lead to a considerable reduction of life expectancy and quality of life. The BSH welcomes this world first by the UK government to help the sickle and thalassaemia communities
UK haematologists across the country care for a large population of patients with these conditions and are excited by the prospect of using this cutting-edge therapy which appears to offer an effective cure in those treated within the clinical trials.
We are hopeful that NICE will approve this potentially life-changing therapy in both sickle cell and transfusion-dependent thalassaemia making the treatments as accessible as possible.