ASH and FDA Unveil New Recommendations to Guide Clinical Development of Sickle Cell Disease Therapies

Sickle cell disease is the most common inherited red blood cell disorder in the United States, and it affects millions of people worldwide. In people living with SCD, the red blood cells, which are normally round, become crescent or sickle-shaped which contributes to the vaso-occlusive crises these patients experience. People with SCD suffer from an array of physical complications, including acute pain crises, joint and organ damage, impaired cognitive function, and a reduced life expectancy. In addition to the immense physical burden they must endure, people with SCD are often stigmatized due to a poor understanding among healthcare professionals and the general public of the life-limiting effects of the disease. Supporting SCD research and access to care efforts for all people living with the disease is a chief priority for ASH.

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