Thalassemia and sickle cell disease (SCD) are disorders of hemoglobin that affect millions of people worldwide. The carrier states for these diseases arose as common, balanced polymorphisms during human history because they possibly afforded protection against severe forms of malaria. These complex, multisystem diseases demand comprehensive, multidisciplinary and lifelong system of care.
While the emphasis should be on prevention, let us try to get an idea as to what medical science has to offer in the near future in terms of management as we commemorate World Thalassemia Day on 8th May. Gene Therapy: On 29th March 2019, the European Medicines Agency (EMA) has recommended granting a marketing authorization in the European Union for a genetically modified product for beta-thalassemia. LentiGlobin vector BB305 is intended for adult and adolescent patients 12 years and older who need…
