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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 .
It is possible for a person to have both the gene for hemoglobin S (the form associated with sickle cell anemia) and the gene for hemoglobin C; this state is called hemoglobin SC disease, and is generally more severe than hemoglobin C disease, but milder than sickle cell anemia. [2]
The commander of the American Expeditionary Force in France in World War I was in a coma and sinking. Mason made the medical report on General Pershing, who recovered and lived for 10 years more. When Howard Hughes lay near death in Los Angeles in 1946, following the crash of his experimental plane, the XF-11, he summoned Mason and asked, "Am I ...
This would be similar to the hemoglobin allele which is responsible for sickle-cell disease, but solely in people with two copies; those with just one copy of the allele have a sickle cell trait and gain partial immunity to malaria as a result. This effect is called heterozygote advantage. [27]
Sickle cell nephropathy is a type of kidney disease associated with sickle cell disease which causes kidney complications as a result of sickling of red blood cells in the small blood vessels. The hypertonic and relatively hypoxic environment of the renal medulla , coupled with the slow blood flow in the vasa recta , favors sickling of red ...
During TLC's first national tour, as MC Hammer's opening act, Lopes and Thomas discovered that Watkins had sickle-cell anemia, a blood disorder which she kept as a closely guarded secret until becoming extremely ill while touring with TLC in the Southwest US, ending up hospitalized, with Lopes and Thomas staying with her, resulting in the ...
It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity. [9] It was developed by Global Blood Therapeutics, a subsidiary of Pfizer. [10] 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.
Although most patients have a decrease of erythropoiesis (production of red blood cells) during parvovirus infection, it is most dangerous in patients with pre-existing bone marrow stress, for example sickle cell anemia or hereditary spherocytosis, [43] [44] and are therefore heavily dependent on erythropoiesis due to the reduced lifespan of ...