The Food and Drug Administration may be one step closer to what could be the first approval of a drug that uses the groundbreaking gene-editing tool CRISPR.
The drug, called exa-cel, treats sickle cell disease, an inherited blood disorder that affects an estimated 100,000 people in the U.S., most of whom are Black, according to the Centers for Disease Control and Prevention.
The illness causes the body’s red blood cells, usually disk-shaped, to take on a crescent or sickle shape. When that occurs, the cells can clump together, leading to clots and blockages in the blood vessels. That may result in a variety of complications, including excruciating pain, trouble breathing or stroke.
Heartfelt and earnest response that makes me feel so shitty there isn’t anything to help alleviate what ails you. Hopefully you’ll get your cure soon enough as well.
I appreciate that, but I honestly care more about them than me. What I have is pretty rare. What they have is far more common. Far more people are suffering. I have medication that helps mitigate the issue and allows me to function basically normally. There are people with sickle cell that can’t get out of bed.