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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.
Younger patients with cystic fibrosis are to benefit from “life-changing” drugs after regulators authorised their use among babies, toddlers and reception-aged children.
A child who received a life-changing cystic fibrosis drug has told how she felt better within hours. Kate Farrer, seven, is one of the first young children to be given Kaftrio on the NHS.
Common chronic illnesses in children include asthma, diabetes, cystic fibrosis, obesity and overweight, malnutrition, developmental disabilities and differences, and mental illness. [7] Leading causes of poor outcomes due to childhood chronic illness, however, depend on geographic region.
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]
Judah, a 14-year-old boy who was diagnosed with cystic fibrosis at just two weeks old was granted a wish by the Make-A-Wish Foundation to meet and record music with Jason Derulo
The Sarah Murnaghan lung transplant controversy began in late May 2013, when the parents of a ten-year-old Philadelphia area girl with cystic fibrosis, Sarah Murnaghan, launched a campaign to change the under-12 policy of the United Network for Organ Sharing (UNOS), which they asserted was age discrimination.
Claire Lucia Wineland (April 10, 1997 – September 2, 2018) was an American activist, author, speaker and social media personality. [2] [3] Through her non-profit organization, Claire's Place Foundation, she provided support to children and families affected by cystic fibrosis (CF).
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