Cystic Fibrosis Foundation

From identifying research priorities to informing clinical care guidelines, Community Voice members play a vital role in shaping CF research, care, and programs. Their dedication and willingness to share their insights and experiences through surveys, focus groups, and committees help drive meaningful change in the CF community. Want to learn more about Community Voice? Explore current projects and discover how you can share your voice: https://lnkd.in/ewHYVy4c

Community Voice provides an opportunity to bring your unique insights and priorities to the forefront of cystic fibrosis research, care, and programs. By collaborating with the CF Foundation, researchers, and clinicians, members help shape a brighter future for the entire CF community. Tijana Milinic, MD, fellow, pulmonary and critical care medicine, University of Washington, shares how members impacted her work. “Partnering with the CF community helped us better understand the experiences of Black, Hispanic, and people of color in CF clinical research. This partnership is an important step toward building a more welcoming and inclusive research environment for all people with CF." Discover how you can share your voice. Explore a selection of our current projects, and join Community Voice today: https://lnkd.in/dtMEzJXm

We have agreed to invest up to $15 million in ReCode Therapeutics to fund the development of a gene editing therapy that is designed to use ReCode’s delivery system to transport gene editing cargo to the lung cells in people with cystic fibrosis. First, the lab research will target the correction of a nonsense mutation. Other rare mutations that are not eligible for modulators will be explored later. “Gene editing is a promising, complex technology, which will take many years to develop into a therapy for people with CF,” said Steven Rowe, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “By investing in this research now, we are laying the foundation to develop what we hope will be a transformative treatment for people with CF, especially those who can’t take CFTR modulators.”

Our Senior Vice President and Chief Policy and Advocacy Officer, Mary Dwight, recently served as a panelist at the 2024 National Organization for Rare Disorders Summit, where she and her fellow panelists discussed the differing perspectives on balancing cost, innovation, and access when trying to achieve a sustainable, affordable health care system for rare disease patients. “The story of cystic fibrosis is the story of passionate advocacy — first and foremost by parents living with the disease and now increasingly adults living with CF — aggressive innovation, and shameless collaboration,” she said. “Our endgame remains a vigorous pursuit of a cure, something that effectively treats everybody with CF and all the ramifications of their disease. Part of this effort is ensuring legislators hear the voice of the patient. We need to ask 'where is the patient' with any legislative effort. What we always look at is whether that legislation or policy will positively impact someone with CF.”

Thank you to everyone in the cystic fibrosis community who participated in our long-term strategic plan survey! Together, nearly 2,000 of you shared your perspectives about how to best support our growing, ever-evolving community. Your feedback will inform the Foundation’s priorities to drive continued progress in cure, care, and community. Results will be shared next year, and the strategic plan will be shared in 2026.

Open enrollment is here! Selecting the right health insurance plan is crucial for people with cystic fibrosis and their families. With free, personalized one-on-one assistance, Compass can help take the guesswork out of choosing the plan that best fits your needs. Even if you plan to keep the same health insurance plan, your coverage could still change. Our dedicated Compass case managers are available in English and Spanish to help review your options and make sure you get the coverage you need in 2025. Compass can even help support other non-English speaking people with CF and their families through interpretation services. Get started today: https://on.cff.org/3NTuphO

Have a question following this year’s North American Cystic Fibrosis Conference? We’ve got answers. Join us November 12 at 7 p.m. ET for a live NACFC highlights presentation and community question-and-answer session with Drs. Cynthia Brown and Dan Dorgan, moderated by Chyann Hoyle, an adult living with cystic fibrosis. Save your seat: https://lnkd.in/gcvg_t6i

Living with cystic fibrosis can be emotionally challenging. To better address and support the mental health of children with the disease, we’ve awarded $3 million to support research focused on the best ways to screen for mental health issues and identify potential risk factors. Led by Beth Smith, a psychiatrist at the University at Buffalo, this new study is an outgrowth of The International Depression Epidemiological Study (TIDES), which was the largest study of mental health in adolescents and adults with CF.

We were thrilled to have so many guests swinging for a cure at our ultimate golf experience, The Drive, in San Diego, California. During the event, we had the honor of presenting the Champion for a Cure award in memory of Sheri Mount, one of the event founders, to an outstanding individual who exemplifies the true spirit of The Drive. This year’s recipient was Beau Guyette, a dad to a person with CF. Along with his wife, Andrea, he has dedicated himself to supporting the Foundation’s mission and growing the event. In addition, please join us in congratulating Katy Burkhart, the Giovanni Villani Champion Award. This award is given in memory of Giovanni Villani, a passionate golfer and person with CF, to the tournament’s top golfer of the year. The recipient receives The Drive’s coveted blue jacket. We thank all our sponsors and supporters who made this event possible. Together, we can end CF.

During this year’s North American Cystic Fibrosis Conference, we were honored to give the Carolyn and C. Richard Mattingly Leadership in Mental Health Care Award to Dr. Mary Beth Prieur, MD, licensed psychologist and associate professor at the University of North Carolina at Chapel Hill. The award is given by the Foundation each year to a member of the cystic fibrosis community in recognition of their leadership and commitment to the mental health and well-being of individuals with CF. Mary Beth has demonstrated unwavering dedication to improving the mental health of children with CF for the past 14 years, and her innovative approaches to addressing mental health challenges have made a significant impact on the CF community. She coaches her CF care team through difficult conversations with children and families, helping them to better understand how to assess needs and provide support. As a member of the CF Foundation’s mental health advisory committee, she has expanded her influence by coaching other CF care teams and working on numerous mental health tip sheets for CF clinicians. Mary Beth is a leader, educator, and researcher with a wonderful sense of humor appreciated by all who know her. She never lets her team forget that kids with CF are kids first and their lives are much bigger than their disease.