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For the first time in history, there’s hope that the debilitating illness known as sickle cell disease may be treatable — even cured.
The Food and Drug Administration announced Friday that two gene therapies for sickle cell disease have been approved following years of clinical trials.
Sickle cell disease is an inherited blood disorder caused by a mutated gene that affects hemoglobin, a protein in red blood cells, according to Johns Hopkins Medicine.
Because those crescent or sickle-shaped blood cells can’t effectively carry oxygen through the body, people with sickle cell disease face a lifetime of organ failure, anemia, strokes and excruciating pain.