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The Cystic Fibrosis Foundation and Vertex Pharmaceuticals of Cambridge, Mass. have announced a successful trial for a new drug that treats the underlying cause of cystic fibrosis.
About 30,000 people in the United States have CF, which is caused by a genetic mutation that creates a buildup of thick mucus in the lungs and digestive system. People with CF rarely live beyond their mid-30’s. Drugs currently on the market only treat symptoms of the disease, while the new medication targets the defective protein that causes it.
Patients in the study experienced dramatic improvements in lung function and weight gain. We speak with one of them, Emily Schaller of Detroit, who is breathing better than she can ever remember.