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DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...
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 ...
Sickle cell disease (SCD), also simply called sickle cell, is a group of hemoglobin-related blood disorders typically inherited. [2] The most common type is known as sickle cell anemia. [2] It results in an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. [2] This leads to a rigid, sickle -like shape under ...
The agency also approved another more traditional gene therapy for the disease as well: lovo-cel (brand name: Lyfgenia) from bluebird bio, giving sickle cell patients two powerful new ways of ...
Victoria Gray. 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. [1]
Hematopoietic stem cell transplantation. Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood, in order to replicate inside a patient and produce additional normal blood cells. [1][2][3][4][5][6] HSCT may be ...
"One of the latest statistics I have seen is that SCD affects approximately 100,000 Americans. Most are African American," says columnist Sharon West.