Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.
Yesterday, President Donald J. Trump signed the Right to Try bill into law. The House approved the bill last week and the Senate passed it last summer.
The Patient Registry is an invaluable tool for researchers conducting observational studies about people with CF in the U.S. About 50,000 individuals have been followed in the Registry since its inception in 1986; many of them have been included for over 20 years.
The Success with Therapies Research Consortium helps the clinical study of interventions so people with cystic fibrosis can improve the consistency of their day-to-day management of their CF to optimize health outcomes and quality of life.
The Cystic Fibrosis Foundation recently signed on to a letter with more than 70 organizations in opposition to the Right to Try Act, which passed in the House of Representatives earlier this month.