Hemoglobinopathies in the Fetal Position

(N Engl J Med. 2018;379:1675–1677) Hemoglobinopathies are disorders caused by mutations in genes that encode globins and include sickle cell disease and β-thalassemia. It has been reported that adults with these hemoglobinopathies who have comparatively high levels of fetal hemoglobin (HbF) have better clinical outcomes than those with low levels of HbF. While drugs like hydroxyurea increase HbF levels, thereby reducing the severity of symptoms of hemoglobinopathies, a drug that more effectively increases HbF levels is the holy grail of hemoglobinopathy research.