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Sunday, January 27, 2019

These Patients Had Sickle-Cell Disease. Experimental Therapies Might Have Cured Them. - The New York Times


A slide of blood showing abnormal red blood cells (sickle cells). Note the bizarre shapes. Normal red blood cells are round or spherical.  This is caused by abnormal hemoglobin which interferes with the transportation of oxygen to the cells of the body.

Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain.  


Employment can be difficult because the disease is debilitating. Yet many who apply for Social Security disability are denied, Dr. Tisdale said. They end up at emergency rooms when they are in crisis.
And treating the disease, with its complications, is expensive: annual costs per patient are estimated at $10,000 a year for children and $30,000 for adults. Those with the disorder go in and out of hospitals.

Tantalizing science



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