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Berkeley Lovelace Jr. and Marina Kopf. December 8, 2023 at 10:19 AM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness...
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.
Federal officials Friday approved two gene therapy treatments for sickle cell disease, welcome news for thousands with the inherited blood disorder that causes terrible pain crises and...
The care of people with sickle cell disease may include infection prevention with vaccination and antibiotics, high fluid intake, folic acid supplementation, and pain medication. [5] [6] Other measures may include blood transfusion and the medication hydroxycarbamide (hydroxyurea). [6] In 2023, new gene therapies were approved.
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.
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 ...
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.
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 ...
The independent committee is helping the FDA think through how it should evaluate a treatment called exa-cel that could potentially cure people of sickle cell disease, a painful and deadly disease ...
Sickle cell trait describes a condition in which a person has one abnormal allele of the hemoglobin beta gene (is heterozygous ), but does not display the severe symptoms of sickle cell disease that occur in a person who has two copies of that allele (is homozygous ).
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