Two Italian women in their twenties on Tuesday received pioneering gene therapy for rare hereditary blood diseases in Pavia in a first for Italy and Europe.
At the San Matteo Hospital in the city south of Milan, the two, respectively affected by β-thalassemia major and severe sickle cell anemia, received for the first time in Italy and in Europe, outside of clinical trials, the advanced gene therapy "Casgevy".
The innovative treatment represents a turning point in the treatment of hemoglobinopathies, serious and disabling genetic diseases.
β-thalassemia and sickle cell anemia are serious hereditary hemoglobinopathies, caused by mutations in the β-globin gene.
