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The FDA is reviewing a new drug for sickle cell disease that uses CRISPR to edit DNA. Black patients with the disease are excited and hopeful about the treatment.
The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [9] [10] [11] It was approved in the United States for the treatment of sickle cell disease in December 2023 and for the treatment of transfusion-dependent beta thalassemia in January 2024. [8 ...
Diamond–Blackfan anemia (DBA) is a congenital erythroid aplasia that usually presents in infancy. [3] DBA causes low red blood cell counts (), without substantially affecting the other blood components (the platelets and the white blood cells), which are usually normal.
Clinical trials of gene therapy for sickle cell disease were started in 2014. [223] [224] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
Treatment depends on the type and cause of the hemolytic anemia. [2] Symptoms of hemolytic anemia are similar to other forms of anemia (fatigue and shortness of breath), but in addition, the breakdown of red cells leads to jaundice and increases the risk of particular long-term complications, such as gallstones [4] and pulmonary hypertension. [5]
Leaky prosthetic heart valves and other cardiac assisted devices can lead to microangiopathic hemolytic anemia (with schistocyte formation) and thrombocytopenia. The force from the blood flow over the high pressure gradient from the prosthesis leads to fragmentation of red cells, and schistocyte formation.
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