Jonathan Sorof, MD, Senior Vice President, Head of Medical Affairs at Global Blood Therapeutics (GBT), talks about the newer treatment options that are now available for persons with sickle cell disease. 

Sickle cell disease is a rare hematologic condition and early symptoms usually occur in childhood and can include swelling of hands and feet; anemia symptoms such as fatigue; and jaundice. As the disease progresses, it can lead to infections, delayed growth, and periodic episodes of pain. Over time organ damage can occur, creating problems in the spleen, brain, eyes, lungs, liver, heart, kidneys, penis, joints, bones, or skin. 

Last November, the FDA approved GBT’s voxelotor which specifically inhibits sickle hemoglobin polymerization, the direct cause of sickle cell disease. The drug was approved for patients 12 years and older but recently the company has announced a plan to seek expanded labelling for patients ages 4 to 11. Previously, blood transfusions were the primary treatment option for sickle cell disease, and many patients still require these transfusions as part of their treatment.

To learn more about this and other rare hematologic conditions, visit


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