A “groundbreaking” cystic fibrosis therapy could profoundly improve patients’ quality of life, say doctors.
Patients often die before their 40s as mucus clogs and damages their lungs and leaves them prone to infection.
A major trial on 1,108 patients, in the New England Journal of Medicine, showed a combination of drugs could bypass the genetic errors that cause the disease and may increase life expectancy.
The Cystic Fibrosis Trust said it could “improve the lives of many”.
One in every 2,500 babies in the UK has cystic fibrosis.
Errors in sufferers’ DNA – inherited from their parents – damage the microscopic machinery that controls salt and water levels in the linings of the lungs.
The result is a thick mucus that inexorably damages the lungs.
Antibiotics help prevent infection and drugs can loosen the mucus, but nothing deals with the fundamental problem for most patients.
The combination of drugs – lumacaftor and ivacaftor – were designed to repair that microscopic machinery.
Susanna McColley, professor of paediatrics at Northwestern University, said these were “groundbreaking findings” that showed the future of treating cystic fibrosis.
She told the BBC: “For subjects I’ve cared for, they felt better in ways that are not necessarily measurable.
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