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Berkeley Lovelace Jr. and Marina Kopf. 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...
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.
Beyond the impact to one’s health, the lifetime cost of treating a patient with sickle cell disease can be $4 to $6 million — despite a median age of death of only 45 years old.
D12765. 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 ...
114,800 (2015) [8] 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]
Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients. It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity. It was developed by Global Blood Therapeutics, a subsidiary of Pfizer.
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