18 September 2025 - Italy has the largest population of people living with transfusion-dependent beta thalassaemia in Europe.
Vertex Pharmaceuticals announced today a reimbursement agreement with the Italian Medicines Agency for eligible transfusion-dependent beta thalassaemia and severe sickle cell disease patients to access the CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel).