For the estimated 1,500 Americans suffering from a blood disorder known as beta thalassemia, the Food and Drug Administration's approval Aug. 17 of the drug Zynteglo was almost unalloyed good news.
As a scientific achievement, Zynteglo is remarkable. The treatment has been shown to effectively cure the disorder in as many as 90% of test cases.
That's a life-changing development for those with the inherited condition, many of whom are otherwise condemned to receiving blood transfusions as often as every two weeks. The median age of death of those with beta thalassemia, which is typically diagnosed within a few weeks of birth, is 37.
