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


The American Society of Hematology (ASH) today released the most comprehensive set of recommendations to date aimed at establishing uniformity and global standards for clinical trial endpoints used to evaluate new therapies for sickle cell disease (SCD). The new recommendations – published in two companion papers in the current issue of Blood Advances – are the result of seven expert and patient led panels convened by ASH and the U.S. Food and Drug Administration (FDA) to improve the design of clinical trials for new SCD therapies, including promoting broader use of patient reported outcomes and biomarkers as clinical endpoints.

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.

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