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An independent review board has found Global Blood Therapeutics’ sickle cell disease treatment voxelotor safe, and recommended that its Phase 3 clinical trial continue.
The hallmark of sickle cell disease is damage to a red blood cell protein called hemoglobin that carries oxygen to organs and tissue. The damage causes the cells to become shaped like sickles rather than having a normal disc shape. The sickling interferes with hemoglobin’s ability to deliver oxygen to tissue.
Global Blood designed voxelotor to increase sickle-shaped hemoglobin’s ability to carry oxygen. The primary objective of the 24-week, Phase 3 HOPE trial (NCT03036813) is to see whether voxelotor does increase sickled hemoglobin’s ability to transport oxygen. Patients have been divided into two groups. One is receiving 900 mg of the therapy, and the other 1,500 mg.
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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.