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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 ...
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a patient’s ...
The two gene therapies are the first approved in the U.S. for sickle cell disease. The FDA has previously OK’d 15 gene therapies for other conditions. In the U.S., an estimated 100,000 people ...
The Centers for Disease Control and Prevention says 100,000 Americans are living with sickle cell disease. FDA approves first gene therapies to treat sickle cell disease Skip to main content
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 ...
D11930. Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia. [ 1 ][ 5 ][ 2 ] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015. [ 6 ][ 7 ] The most common adverse reactions include reduced ...
The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life. ... Although not enough time has passed to ...
Voxelotor. Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. [1][3][4][5][6] Voxelotor is the first hemoglobin oxygen-affinity modulator. [7] Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell ...
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