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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...
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 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.
When treating avascular necrosis of the bone in people with sickle cell disease, the aim of treatment is to reduce or stop the pain and maintain joint mobility. Current treatment options include resting the joint, physical therapy, pain-relief medicine, joint-replacement surgery, or bone grafting.
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 ...
Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease. Voxelotor is the first hemoglobin oxygen-affinity modulator. Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.
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 ...
Transfusion therapy for sickle-cell disease entails the use of red blood cell transfusions in the management of acute cases of sickle cell disease and as a prophylaxis to prevent complications by decreasing the number of red blood cells (RBC) that can sickle by adding normal red blood cells.
The FDA approved exa-cel for sickle cell disease, and will make a decision about treating beta thalassemia by March 2024.
Hydroxycarbamide, also known as hydroxyurea, is a medication used in sickle-cell disease, essential thrombocythemia, chronic myelogenous leukemia, polycythemia vera, and cervical cancer. In sickle-cell disease it increases fetal hemoglobin and decreases the number of attacks. It is taken by mouth.