Study Offers Hope To Patients Suffering From Genetic Disease Cystic Fibrosis

According to statistics, around 8,000 people in the UK suffer from the genetic disease cystic fibrosis (CF). CF produces thick, sticky mucus that clogs the lungs and the pancreas, leading to life-threatening chest infections and problems with digestion presently there is no cure for this disease. There are medications to ease symptoms of this disease but currently there are no medications to cure it fully.

The recent research by the researchers at the University of Bristol has offered a ray of hope to patients suffering from CF. They have developed a new drug to cure cystic fibrosis. Presence of CF can be detected by 'salty sweat', which is caused by the failure of the sweat ducts to reabsorb sweat.

In the clinical trails of this drug, the patients who were given 150mg twice a day saw the concentration of salt in their sweat decrease by almost 50% and lung function improve by 10%.

Study author, Dr. David Sheppard from the University of Bristol said: "The early results with VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients."

According to statistics, one in 25 people in the UK is a carrier for a defective CF gene which disables or destroys a protein known as CFTR. Hence the defects in the CFTR protein affect the transport of chloride and other ions across cells. The study showed that VX-770 is designed to increase the probability that the CFTR channel is open, thereby increasing chloride transport across the cell surface.

Till now researchers have tested effect of this medication on CF patients with a genetic defect known as G551D. Researchers added that more clinical trails are required to test benefits of this drug on other patients.

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