Since scientists pinpointed the gene responsible for cystic fibrosis in 1989, the nonprofit group has recruited study participants and donated millions of dollars to develop gene-based treatments. In January the Food and Drug Administration (FDA) approved Kalydeco, the first drug to treatpeople with a mutation of that gene. Kalydeco will help only about 4 percent of sufferers of the chronic lung disease, but its approval is an important step toward developing personalized medicines.
展开▼
