Hemoglobinopathy are a group of disorders that are inherited, in which there is abnormal production or structure of the hemoglobin molecule. Hemoglobinopathy is the medical term for a group of inherited blood disorders and diseases that primarily affect red blood cells. Sickle cell disease (SCD) is one such blood disorder caused by the abnormal hemoglobin that damages and deforms red blood cells. There are two main groups, which are abnormal structural hemoglobin variants caused by mutations in the hemoglobin genes, and the thalassemias, which are caused by an alteration in normal hemoglobin molecules. The main structural hemoglobin variants are HbS, HbE and HbC. The main types of thalassemia are alpha-thalassemia and beta thalassemia. The two conditions may overlap because some conditions, which cause abnormalities in hemoglobin proteins also affect their production.
Some hemoglobin variants do not cause pathology or anemia, and thus are often not classed as hemoglobinopathies. Common symptoms of the condition include, severe pain, shortness of breath, enlarged spleen, and growth problems in children. It can be any group of disorders caused by the presence of variant hemoglobin in the red blood cells.
Market Dynamics
Increasing adoption of organic growth strategies, such as partnerships by key players in the market to expand their product portfolio, is expected to drive the market growth over the forecast period. For instance, in June 2022, Precision BioSciences, a publicly traded American clinical stage gene editing company, and Novartis AG, a pharmaceutical corporation, announced that they have entered into a partnership to develop new gene therapy for blood disorders.
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