New drug offers hope to cystic fibrosis sufferers

Breakthrough pill first to target genetic cause of disease rather than symptoms

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Cystic Fibrosis (CF) suffers have been offered new hope in the form of breakthrough drug Kalydeco.

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The medication is the first of it’s kind, targeting the genetic cause rather than the symptoms of the disease, and could help the 360-400 CF sufferers in the UK with the G551D genetic mutation.

It works by correcting the defective protein CTFR so salt and fluids can flow properly over the surface of the lungs.

The drug has been available in the US since February with promising results.

Iva Caftor Patient Interest Group has teamed up with the Cystic Fibrosis Trust to persuade Parliament to debate whether Kalydeco should be available on the NHS, but need 100,000 petition signatures to do so.

CF is one of the UK’s most common life-threatening diseases in children and young adults.

A faulty gene causes the disease and makes the internal organs, particularly the lungs, become clogged with thick and sticky mucus, resulting in infection. 

“Every single day a baby is born with CF. Despite the best treatment, they’re unlikely to make it past their 30th birthday,’ said TV personality Amanda Lamb in support of the campaign.

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“This drug will allow these babies to live a longer, more normal life.”

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