EIN 51-0169988

2024 Education Highlights

• Hosted the 37th National CF Education Conference as a hybrid event, where 30 speakers – including renowned experts in the field of CF, patient panelists, and CF researchers – presented the latest in research and clinical practice to over 100 in-person attendees, and to over 400 virtual attendees from our global CF community. Evaluations found that over 95% of participants improved their knowledge of CF and treatment options.

• Distributed nearly 10,000 CFRI-created informational brochures and booklets available in English and Spanish, including CF in the Classroom, A Teen’s Guide to Navigating Cystic Fibrosis, Patient Assistance Resources, and CF and Cancer Awareness.

• Produced and posted 21 podcasts through our CF Community Voices podcast series, with over 2,000 combined views.

• Maintained our website as a dynamic resource for the global CF community. In addition, CFRI engaged its 19,000+ Facebook, Twitter, LinkedIn, and Instagram followers with breaking news, resources, and inspiring stories.

• Distributed 18,000 copies of the CFRI Community newsletter to homes and health centers across the globe. Distributed 6,000 copies of CFRI Comunidad, CFRI’s Spanish-language newsletter, to CF centers nationwide.

• Provided updates on CF research, special events, advocacy efforts, and support programs through our weekly eNewsletter, distributed to over 15,000 subscribers.

2024 Support Highlights

• Provided nine monthly facilitated virtual Support Groups with over 600 participants from across the U.S. and globe. Groups are provided to: adults with CF; caregivers of children with CF; parents/spouses/partners of adults with CF; people who lost a loved one to CF; adults with CF post-transplant; teens with CF; Spanish-speaking CF community members; adults with a late CF diagnosis; those who cannot use modulators.

• Underwrote individual therapy sessions for 85 individuals with CF and/or their immediate family from 20 states, resulting in a statistically significant reduction in depression and anxiety symptoms.

• Offered free weekly online CF-specific wellness classes, including strength training, yoga, pilates, and dance, attended by over 100 CF community members.

• Hosted four retreats for the national and global CF community. These included a virtual CF Spring Retreat and hybrid Summer Retreat for adults with CF and an in-person Spring Retreat and virtual Fall Retreat for mothers of children/adults with CF. These events provided resources, research updates, psychosocial support, and connection to peers; evaluations showed measurable decreases in symptoms of depression and anxiety.

• Increased support for the Spanish-speaking CF community via print, social media, and website resources, including a Spanish language version of CFRI’s newsletter.

2024 Advocacy and Awareness Highlights

• Participated in 45 virtual and in-person meetings with state and federal legislators and policy makers regarding issues impacting the health and quality of life for those with CF, incl. access to care.

• Pursued efforts to create state Rare Disease Advisory Councils (RDACs) through coalition building with rare disease groups. Led efforts to pass legislation to create an RDAC in California.

• Advanced awareness of the racial and ethnic diversity of the CF community through the creation of podcasts and films. Expanded informational sheets in multiple languages; expanded Hindi and Spanish language offerings on our YouTube channel; provided captioning and subtitles for conference attendees; made CFRI website accessible in multiple languages.

• Engaged with other disease groups to advocate for pharmacy benefit manager (PBM) reform, copay accumulator bans, the prohibition of co-pay accumulator programs and surprise medical billing, while raising awareness of potential risks posed by state Prescription Drug Affordability Review Boards (PDABs) to CF community access to therapies.

• Produced an impactful CF Awareness and Advocacy video featuring members of the CF community to inspire greater engagement in advocacy activities.

• Partnered with other rare disease groups to engage our CF community on issues that impact the rare disease community. Sent multiple action alerts to 15,000 community members, facilitating communication between them and their representatives.

• Participated in diverse activities to raise awareness of the discriminatory use of Quality Adjusted Life Year (QALY) to assess drug pricing and value for rare disease drugs.

• Continued alliances with our CF organizational partners in the Cystic Fibrosis Engagement Network, with national rare disease organizations, disability groups, and with our partner organizations in the American Thoracic Society Public Advisory Roundtable.

2024 Research Highlights

In 2024, the following researchers conducted research with the support of CFRI:

Elizabeth Nash Memorial Fellowship Program

Steven Jonas, MD, PhD (Principal Investigator), Ruby Sims, PhD (Postdoctoral Fellow) – University of California Los Angeles Designing a Cystic Fibrosis Gene Therapy Nanocarrier Platform to Target and Modify Airway Stem Cell-Derived Ionocytes

Ron Kopito, PhD (Principal Investigator), Celeste Riepe, PhD (Postdoctoral Fellow) – Stanford University Pharmacogenomic Discovery of Therapeutic Targets for Corrector-Resistant Cystic Fibrosis

Matthew Porteus, MD, PhD (Principal Investigator), Anais Amaya Colina, PhD (Postdoctoral Fellow) – Stanford University A Hybrid Gene Correction Strategy for Cystic Fibrosis

New Horizons Program

Nadia Ameen, MD – Yale University School of Medicine Elucidating The Ion Transport Functions of CFTR High Expresser Cells (CHEs) And Its Relevance To Intestinal Disease In Cystic Fibrosis

Benjamin Chan, PhD – Yale University School of Medicine Optimization of Phage Therapy to Reduce PseudomonasInduced Inflammation in Cystic Fibrosis

Daria Van Tyne, PhD – University of Pittsburgh Optimization Of Activity and Improved Delivery of Bacteriophages Targeting Burkholderia Spp

Katrine Whiteson, PhD – University of California Irvine Targeting Recalcitrant CF Pathogens with Phages, Antibiotics, And Small Molecule Adjuvants

Feng Yuan, PhD – University of Alabama at Birmingham Dissecting Pulmonary Ionocyte Subtypes and Their Functional Roles in Cystic Fibrosis