CFTR Modulator Therapies
CFTR Modulator Therapies
Aug 13, 2024
cff.org
Types of CFTR Mutations
Types of CFTR Mutations
Oct 16, 2024
cff.org
Since 2012 when the first CFTR modulator, Kalydeco, was approved, modulator therapy has transformed cystic fibrosis care. By the end of 2024, 24,524 people with CF in the United States were prescribed Trikafta, according to data from the Patient Registry. However, 7.3% of people with CF were still not eligible for any CFTR modulators due to their age or genotype. For them, our work is not done. We are committed to finding a cure and advancing therapies for every person with CF. | Cystic Fibrosis
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Since 2012 when the first CFTR modulator, Kalydeco, was approv…
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The journey to end cystic fibrosis isn’t a straight line. It’s an evolving map with many paths and unique challenges. That’s why we’re pursuing several different approaches to help achieve our goal of restoring CFTR protein and function in 100 percent of people living with cystic fibrosis. Dr. Bill Skach, EVP and Chief Scientific Officer at the Foundation, discusses how genetic-based therapies such as mRNA therapy, DNA therapy or gene therapy, and gene editing, could allow normal CFTR protein to
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