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Cystic fibrosis (also known as CF or mucoviscidosis) is an autosomal recessive genetic disorder affecting most critically the lungs, and also the pancreas, liver, and intestine.
In December 2020, after an additional clinical trial was completed, and FDA approval was expanded for 177 other cystic fibrosis mutations. [32] FDA approval for children aged 6–11 was added in January 2021, after a third clinical trial was completed. [33] In 2023, approval was extended to children 2–5. [34]
Cystic fibrosis is an inherited (genetic) disease that can present with symptoms within the first two years of life. The genetic defect results in the production of thick mucus in the lungs, pancreas, liver, small intestine, and reproductive organs.
Younger patients with cystic fibrosis are to benefit from “life-changing” drugs after regulators authorised their use among babies, toddlers and reception-aged children.
Fibrosing colonopathy is a disease that arises in people with cystic fibrosis treated with high doses of pancreatic enzyme supplements. [1] [2] Symptoms are non-specific with abdominal pain, abdominal swelling, vomiting, and constipation. [1]
Differentiation of DIOS from constipation is generally performed by a unit specializing in the treatment of cystic fibrosis. Adequate hydration and an aggressive regimen of laxatives are essential for treatment and prevention of DIOS. Osmotic laxatives such as polyethylene glycol are preferred. [1]
14-year-old Judah was diagnosed with cystic fibrosis at 2 weeks old and with the help of Make-A-Wish, he was able to make his musical dreams come true
Cystic fibrosis (or CF for short) is a genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestines. [ 1 ] [ 2 ] Symptoms