Cystic Fibrosis is a disease-causing mutation in the CFTR gene which impairs the transport of chloride ions and the movement of water into and out of cells. As a result, cells that line the passageways of the lungs, pancreas, and other organs produce mucus that is abnormally thick and sticky causing obstructions in the airways and glands.
Just recently, the U.S. FDA Accepted Priority Review for the Use of Trikafta® in Children with Cystic Fibrosis Ages 6 to 11 who have at least one copy of the F508del Mutation set for June of this year. This third modulator medication could potentially get Fawkes’ CFTR function to acceptable levels. The medicine was funded by the Cystic Fibrosis Foundation for Vertex to invent. But as everyone knows drug R&D costs are very expensive. The foundation still helps patients access this and other life-saving medications and treatments.
If you'd like to learn more about my and Fawkes' journey with CF, you can visit my blog where I documented his medications, therapies, and other adventures. http://fawkespatrol.blogspot.com/
Will you join us?
Great Strides is a fun event that provides a fantastic opportunity for family, friends, students, and colleagues to come together to make a difference in the lives of people with CF.
To participate, just click on the "Join our Team" button. From there, you can make a donation and start fundraising.
By joining our Great Strides team and making a donation, you will be part of a tenacious and passionate group of people committed to ending this disease. Together, let’s make CF stand for Cure Found!
IMPORTANT NOTE ON ATTENDANCE AT FOUNDATION EVENTS:
Updated July 5th, 2023
The CF Foundation is committed to ensuring the health and wellbeing of individuals attending Foundation events. Individuals attending CF Foundation events must abide by the Foundation's Event Attendance Policy www.cff.org/attendancepolicy, which includes guidance for event attendees living with cystic fibrosis.