Medication that targets genetic mutation too expensive for the NHS
A breakthrough drug has offered new hope for some sufferers of the lung disease cystic fibrosis (CF).
Kalydeco, the first medication to target the genetic cause rather than the symptoms of the disease, could help the 360-400 CF sufferers in the UK with the G551D genetic mutation.
The drug corrects the defective protein CTFR so that 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 been working 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.
The disease is caused by a faulty gene that makes the internal organs, particularly the lungs, become clogged with thick and sticky mucus which results 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.
“This drug will allow these babies to live a longer, more normal life.”
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