Cystic fibrosis is generally recognized as the most common, fatal genetic illness in the United States; affecting approximately 30,000 people in the U.S. and 70,000 people worldwide. Due to scientific and technical advances, it is also functionally curable within the next generation.
While the genetic underpinnings of CF are more complex than was initially understood, it is still a disease caused by identifiable mutations in a single gene (the Cystic Fibrosis Transmembrane Regulator (CFTR) gene). Therefore, in the landscape of genetic disorders, CF can be thought of as “low hanging fruit” because it represents a disease for which effective treatments may be practically achieved using currently available scientific methods. The CFTR gene was one of the first disease-causing genes discovered by science. CF will also likely be one of the first genetic diseases to be cured.
Thus, a great deal of funding and a substantial proportion of available research hours have been invested in developing treatments for CF. This has produced a substantial and growing body of scientific literature, which continues to accumulate on a daily basis. The purpose of this website is to compile and summarize as much of the leading edge scientific research as possible, and to report on how to access the best available medical care.
Most Recent Research Reports / Abstracts / Papers
- CFTR Modulation Research funded by CFF
- Interim Data from Clinical Trial Support MS1819 as Potential Therapy for EPI in CF Patients
- Enrollment Underway in CF Clinical Trial for Ear Toxicity Caused by Antibiotics
- ProQR’s Inhaled RNA Therapy Improves CF Symptoms in Phase 1 Clinical Trial
- CF Patient, Recovering from Double Transplant, Inspires ‘Lunges4Lungs’ Fundraiser