ivacaftor

Prof Bell, of the Queensland Children's Medical Research Institute, said while it was too early to call ivacaftor a cure as long-term studies were needed, it was "a first step along that path".

NEWS.com.au | Top Stories 2011

Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year, a price roughly in line with those of some other drugs for extremely rare diseases.

NYT > Home Page By ANDREW POLLACK 2012

The mutation targeted by ivacaftor is known as GFF1D, the second most common mutation shared by Australian cystic fibrosis patients.

NEWS.com.au | Top Stories 2011

One of the Australian scientists involved in trials of ivacaftor, Associate Professor Scott Bell, said about 8 per cent of the 3000 cystic fibrosis patients in Australia could potentially benefit.

NEWS.com.au | Top Stories 2011

Half the group was given one ivacaftor tablet twice a day for 48 weeks while the remainder received a placebo.

NEWS.com.au | Top Stories 2011

Published in the New England Journal of Medicine, the results of the phase III trial using the drug ivacaftor showed it could improve lung function by acting against the defect which causes the disease.

NYDN Rss AFP RELAXNEWS 2011

Published in the New England Journal of Medicine, the results of the phase III trial using the drug ivacaftor showed it could improve lung function by acting against the defect which causes the disease.

NYDN Rss AFP RELAXNEWS 2011

Made by the Massachusetts-based Vertex Pharmaceuticals, Kalydeco ivacaftor was approved by the Food and Drug Administration on a fast-track for drugs that may offer big advances over standard therapy.

NYDN Rss 2012

Published in the New England Journal of Medicine, the results of the phase III trial using the drug ivacaftor showed it could improve lung function by acting against the defect which causes the disease.

NYDN Rss AFP RELAXNEWS 2011

For the ivacaftor trial, 19 of the 161 patients who took part were from Australia.

NEWS.com.au | Top Stories 2011