Among the candidates listed for lung transplant, those with an indication of CF decreased from 13.3% in the pre–highly ...

The genetic condition cystic fibrosis had no treatments on the NHS before 2019 and a Daily Express campaign - now a miracle ...

The Hospital for Sick Children. "Newly uncovered mechanism could drive next-gen cystic fibrosis treatments." ScienceDaily. ScienceDaily, 13 March 2025. <www.sciencedaily.com / releases / 2025 / 03 / ...

A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...

Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator ...

The past decade has been a game-changing one for Vertex Pharmaceuticals (NASDAQ: VRTX). The biotech won approval for its ...

UK MHRA approves Vertex’s Alyftrek, a once-daily next-in-class CFTR modulator to treat cystic fibrosis: London Monday, March 10, 2025, 10:00 Hrs [IST] Vertex Pharmaceuticals, a ...

The agency approved it based on two Phase III studies showing the drug to be as effective as another combination therapy at improving lung function.

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today approved Alyftrek (triple combination medicine ...

Vertex announced that the United Kingdom Medicines and Healthcare products Regulatory Agency has granted approval for ALYFTREK, a once-daily ...

In two randomised phase 3 studies involving 480 participants aged 12 years and over, ivacaftor/tezacaftor/vanzacaftor was ...

I got dangerously sick while waiting for double-lung and liver transplants. But then I got a second chance at life after starting Trikafta, even though I have two rare mutations.