The U.S. Food and Drug Administration approved Kalydeco® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.
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My wife and I were heartbroken when our daughter, Ruth, was diagnosed with cystic fibrosis as a newborn. But CF can’t stop us from being optimistic about what the future holds for our family.
Advances in cystic fibrosis care have been life-changing for me, and I’m grateful to breathe easier with a CFTR modulator. However, I began to experience new issues in the past few years, and I am left wondering if it’s a side effect of the CFTR modulator or something else.
Cystic fibrosis defined my identity for many years until I participated in clinical trials for Trikafta®. As my health improved, I struggled to figure out what this meant for my sense of self. This experience motivated me to help other young adults with CF navigate the many changes that modulators can bring.
The Board’s review put Coloradans with CF at the center of the discussion, helping to preserve vital access to Trikafta®.
In comments provided to the U.S.
CF Foundation provides comments to the U.S. Department of Health and Human Services and the Centers for Medicare and Medicaid Services responding to the 2024 Medicare Physician Fee Schedule, focusing on improving access to care through telehealth.
Partnership to Protect Coverage provides comments to the U.S. Department of Health and Human Services, the Treasury Department, and the Department of Labor on a proposed rule concerning short-term, limited-duration insurance plans.
Partnership to Protect Coverage response to request for information issued by the Treasury Department, the Consumer Financial Protection Bureau, and the Department of Health and Human Services on medical credit cards, loans, and other financial products used to pay for health care that may contribute to medical debt.
The Cystic Fibrosis Foundation and the Cystic Fibrosis Therapeutics Network sent a letter to the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research to request a meeting to discuss novel approaches to clinical trial design for cystic fibrosis nucleic acid-based therapies.