Before starting Kalydeco™, the first oral drug that treats the underlying cause of cystic fibrosis, medical student William Elder Jr. had lost most of his sense of smell and suffered from debilitating sinus headaches due to the disease. His lung function was slowly beginning to decline, leading to serious lung infections. The drug changed his life.
“The speed with which Kalydeco was approved has completely changed my life. If it had taken even a year longer, I would have had a much more difficult time staying healthy as a first-year medical student,” the Stanford University graduate said in testimony before a congressional briefing in July.