U.S. regulators have approved a new sickle cell disease medicine that can prevent extremely painful, dangerous flareups in which misshaped blood cells clump together, blocking blood and oxygen flow.
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Sickle cell disease is one of the most common inherited blood disorders, affecting about 100,000 Americans, most of them black, and about 300 million people worldwide. Andemariam, a former Novartis adviser, said the drug appears to work better the longer patients receive it. The debilitating condition also causes anemia, delayed growth, vision damage and painful swelling in hands and feet, making it hard for some people to maintain jobs or attend school.
In a one-year study of 198 patients, those getting the higher of two Adakveo doses averaged 1.6 pain episodes over that year and 36% had none. A comparison group on placebo averaged three pain episodes that year and 17 percent had none. Adakveo’s side effects included influenza and high fever.
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