Three different three-drug regimens all provided dramatic results against cystic fibrosis, a fatal disease of the lungs and digestive system that afflicts 75,000 people worldwide. “This is just what we dreamed would someday happen,” says Francis Collins, the director of the National Institutes of Health, who led the discovery of the cystic fibrosis gene two decades ago. Another 25,000 can benefit from Orkambi, a Vertex drug combination that is far less effective than Kalydeco. Any of these new drugs, it would appear, could get a benefit similar to Kalydeco’s for 90% of cystic fibrosis patients. The efficacy of cystic fibrosis drugs is measured by something called percent predicted forced expiratory volume in one second (FEV1).

Source: Forbes July 18, 2017 20:15 UTC