A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said.

cross-posted from: https://slrpnk.net/post/9281628

Near the end of last year, the Food and Drug Administration gave two companies authorization to sell gene therapy to people with sickle cell disease — a genetic disorder of red blood cells that causes debilitating pain and other medical problems. An estimated 100,000 people in the United States have sickle cell, most of them Black. People are born with the disease when they inherit the mutated gene for the condition from each parent.

The key thing is that this class of treatment is a cure. Once done with treatment, the expectation is that he’ll produce enough normal hemoglobin to live normally.