Kaftrio miracle: How a new suite of genetically-tuned drugs are challenging cystic fibrosis – and opening door for victims to have children

New drug makes it possible for those with cystic fibrosis to have children
Credit: Chemistry World

Becoming a parent for the first time is a major moment for anyone — but for Laura Gayton, giving birth to a healthy, crying baby boy felt nothing short of a ‘miracle’.

Laura, a swimming teacher from Kettering, Northants, is one of around 10,800 people in the UK with cystic fibrosis (CF), a progressive genetic disease that claims the lives of half of those affected before the age of 40.

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Kaftrio, a triple-drug therapy, directly targets the defective protein, making it work more effectively and thinning the mucus. It helps clear the lungs, allowing patients to breathe easier and reducing the risk of infection.

The drug can help around 90 per cent of CF patients, and in September last year Laura became one of the first phase of 60 patients to be given Kaftrio, which is taken as a pill, twice daily — and ‘almost immediately’ she started to feel better.

In the first year the drug became widely available in the U.S., one healthcare trust reported that 18 women with CF — nearly all of whom were taking the drug — became pregnant, a rate of 16 per cent. By comparison, the U.S. national rate of CF pregnancies in 2014 was just 2.5 per cent.

This is an excerpt. Read the full article here

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