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

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 ...

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 ...

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 ...

H.C. Wainwright analyst Ed Arce lowered the firm’s price target on Arcturus Therapeutics (ARCT) to $60 from $63 and keeps a Buy rating on the ...

A boy with a love of trains has gone behind the scenes at a railway signalling hub as a birthday treat.Zach from Walsall has cystic fibrosis and autism and his family said he developed a fascination ...

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

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

Infants with cystic fibrosis lack crucial “beneficial bacteria” in their gut, according to new research. Scientists say this ...

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