Frameshift Mutation
Frameshift Mutation
2 weeks ago
genome.gov
CFTR Modulator Therapies
CFTR Modulator Therapies
Aug 13, 2024
cff.org
Watch this animation to understand how CFTR modulators work for people with F508del mutations. To learn more about drugs in development to restore CFTR function, visit our drug development pipeline: http://on.cff.org/pipeline | Cystic Fibrosis Foundation
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Watch this animation to understand how CFTR modulators work for pe…
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At just four weeks old, James was diagnosed with cystic fibrosis (CF), and his family would learn, that he is one of only 33 people in the world who have this specific rare CF mutation. Treatment required James to take around 60 pills a day, wear a shake vest and use a vaporizer for about 30 minutes to keep his symptoms at bay, impacting his daily life. When the opportunity came to be part of the clinical trial for an FDA approved drug for CF, it was a no-brainer for James' family. Following the
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