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The only cure for painful sickle cell disease today is a bone marrow transplant. But soon there may be a new cure that attacks the disorder at its genetic source.
Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries the risk of rejection by the immune system, in addition to the difficult process of ...
The only cure for painful sickle cell disease today is a bone marrow transplant. On Tuesday, advisers to the Food and Drug Administration will review a gene therapy for the inherited blood ...
Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
The treatment was approved in the United Kingdom for the treatment of sickle cell disease and transfusion-dependent beta thalassemia in November 2023. [10] [11] [12] 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. [13 ...
Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.
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