A 19-year-old with cystic fibrosis (CF) is aiming to run the London Marathon to raise money for charity and awareness for the ...

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

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

Birth defects can be caused by genetic environmental or combined factors but some can be prevented with the right measures ...

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 ...

Approximately 1,000 new cases of Cystic Fibrosis are diagnosed each year that bring costly treatments to many families. The ...

Vertex Pharmaceuticals (NasdaqGS:VRTX) announced that its cystic fibrosis treatment, ALYFTREK®, had received regulatory approval from the UK's MHRA, a significant milestone in expanding its market ...

The genetic disorder cystic fibrosis is unique because it can cause both breathing and digestive problems. Some 40,000 people in the U.S. currently have it, "though this is likely an ...

About 99% of women with cystic fibrosis (CF) use contraception at least once during their fertile years, but unintended pregnancy is common.

Prince Frederik of Luxembourg died on March 1, 2025, at the age of 22 after a prolonged battle with POLG mitochondrial ...

Sionna Therapeutics recently secured an initial public offering (IPO) of $191m on the Nasdaq stock exchange, signalling ...