Thalassemia refers to a heterogenous group of inherited anaemias that are collectively recognised as one of the most common hereditary diseases in the world. Thalassemia is caused by mutations in globin genes leading to a deficiency of haemoglobin synthesis, resulting in ineffective erythropoiesis. The World Health Organization (WHO) has identified thalassemia as a major global health concern and a significant public health challenge. Despite the introduction of preventive and prenatal screening in many countries there is still a significant burden of disease and impact on patient lives. There are several approved treatments including advanced therapies involving novel gene replacement and genome-editing technologies.
Asphalion is proud to support patient groups and sponsors developing new treatment options for thalassemias in order to reduce the cost and improve the overall quality of life.
Can we help? Contact us: [email protected]
