NACFC 2021 | Mariah's Experience with CFTR Modulators

Growing up with cystic fibrosis, Mariah Caise was living a normal childhood. However, in her freshman year of high school, she saw her lung function drop dramatically and was admitted to the hospital with double pneumonia. Soon, her new normal meant being in and out of the hospital, sleeping a lot, and missing classes at school.
In 2019, when Mariah learned her mutations qualified her for a new CFTR modulator, Trikafta, she felt a renewed sense of hope. Today, she has been on Trikafta for nearly two years, and has more energy to participate in sports, had her feeding tube removed, and stabilized her lung function.
Originally recorded September 17, 2021. Featured in plenary 2 of NACFC 2021.

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She’s a beautiful young lady. I’m so glad Trikafta came out that month that it did!