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Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. [8] It initially appeared to have an acceptable safety profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity noted in clinical trials. [9]
In the UK, exagamglogene autotemcel is indicated for the treatment of transfusion-dependent beta thalassemia and sickle cell disease in patients aged 12 years and older who should be treated with hematopoietic stem cell transplantation but for whom a suitable stem cell donor is not available.
The acute chest syndrome is a vaso-occlusive crisis of the pulmonary vasculature commonly seen in people with sickle cell anemia. This condition commonly manifests with a new opacification of the lung(s) on a chest x-ray .
Hemoglobin electrophoresis is a blood test that can detect different types of hemoglobin.The test can detect hemoglobin S, the form associated with sickle cell disease, as well as other abnormal types of hemoglobin, such as hemoglobin C.
Sickle-cell disease was the genetic disorder to be linked to a mutation of a specific protein. Pauling introduced his fundamentally important concept of sickle cell anemia as a genetically transmitted molecular disease. [20] This vein (4) shows the interaction between the malaria sporozoites (6) with sickle cells (3) and regular cells (1).
The Sickle Cell Disease Association of America, Inc. (SCDAA) is a nonprofit organization with the sole purpose of supporting research, education and funding of individuals, families those who are impacted by sickle cell disease.
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