The first drug that treats an underlying cause of cystic fibrosis, a variant called G551D in the CFTR gene, has been approved by the FDA. The drug targets a defect in the protein that causes the disease. This variant is the cause of cystic fibrosis in only 4% of people with the disease, which makes this story useful for teachers using the “Personalized Medicine” lesson, as it illustrates that drugs can be tailored to certain patients based on their genetic information.
A link to the New York Times article is here.
A link to the National Public Radio story is here.