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Latest announcements and gatherings
Sickle Cell Treatment Endari Now Available in the United States
Emmaus Life Sciences has announced that Endari, a medication developed to reduce acute complications in patients with sickle cell disease, is now available by prescription in the United States.
Sickle cell disease is an inherited blood disorder in which the body produces crescent shaped red blood cells that get caught in blood vessels, ultimately blocking blood flow. An altered form of hemoglobin caused by genetic mutations makes red blood cells fibrous and rigid which forms their abnormal shape.
Sickled red blood cells do not last as long as normal red blood cells, causing anemia to develop in patients. Signs that a patient could have sickle cell disease begin in early childhood and include anemia, repeated infections, and periodic episodes of pain, clinically defined as sickle cell crises.
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This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.