http://www.sciencemag.org/news/2019/09/new-sickle-cell-drug-could-soon-get-us-approval-does-it-work

A novel drug may prevent red blood cells from assuming a mutant, sickled shape.

A new drug for sickle cell disease, a grave genetic malady that afflicts an estimated 100,000 people in the United States alone and has no truly effective therapy, should be cause for rejoicing. Instead, what could be a rapid march to approval for voxelotor, which acts directly on the mutant protein that causes the disease, has sparked a dispute over the U.S. Food and Drug Administration’s (FDA’s) efforts to evaluate an urgently needed therapy.