For years, physicians and researchers have been searching for a treatment that helps patients suffering from Diamond Blackfan Anemia (DBA), a bone marrow failure syndrome that affects only 25-35 children per year in America. Since it is resistant to treatments commonly used for anemia, many must rely on glucocorticoid treatments with harsh side effects, with many becoming resistant after prolonged treatment. However, new research has shown that a drug used to lower cholesterol in adults and children actually stimulates a cell receptor that increases red blood cell count in vitro. This study is being used as the foundation for a clinical trial led by experts at the Yale School of Medicine.


Medical Xpress
See article in Nature here