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.
Dr. John P. Clancy shares new developments in drugs that restore airway surface liquid in the lungs of people with cystic fibrosis, making it easier to clear mucus.
Dr. James Chmiel shares new developments in drugs to reduce inflammation in the lungs and help prevent lung damage.
Dr. Jennifer Taylor-Cousar shares new developments in drugs that fight infections in the lungs of people with cystic fibrosis.
For the first time, the Cystic Fibrosis Foundation invited non-CF experts to its annual research conference to meet with CF researchers. The conference -- New Technologies Advancing Toward a One-Time Cure -- in Savannah last month focused on the challenges being faced in gene editing, gene delivery and stem-cell biology and laid the foundation for new collaborations.
Revised Law Includes Designated Funding for Rare Diseases