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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...
While sickle cell disease is most known for causing severe pain, patients can experience stroke, heart disease, organ damage and a dangerous condition called acute chest syndrome caused by ...
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...
Lovotibeglogene autotemcel is indicated for the treatment of people aged twelve years of age and older with sickle cell disease and a history of vaso-occlusive events. [1] [3] The recipient's blood stem cells are genetically modified to produce HbA (T87Q), a gene-therapy derived hemoglobin A, which is similar to the normal adult hemoglobin ...
In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older. [11] [12] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication . [13]
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.
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