A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...

With a market cap of $112.9 billion, Vertex Pharmaceuticals Incorporated (VRTX) is a biotechnology company focused on ...

In a bar, a poet meets the man behind the drug that saved his life — discovering how science, faith and chance created a ...

Modulator pills transformed cystic fibrosis care. But the fight for a cure is not over — so long as research remains funded.

After false starts and incremental gains, scientists crack the problem that made breathing impossible. Will answers come fast ...

Haunted by the cystic fibrosis patients he could not help, one Iowa doctor commits his life to understanding what is broken deep inside their cells.

Patients aged 6 years and older with cystic fibrosis will be able to access the triple combination treatment vanzacaftor/tezacaftor/deutivacaftor on NHS Scotland ...

Cystic fibrosis (CF) is a lifelong genetic condition that primarily affects the lungs and digestive system, making everyday tasks like breathing and eating more difficult than most people ever realize ...

Researchers have demonstrated that lipid nanoparticles can package and deliver an entire therapeutic gene along with gene-editing machinery - a combination of large, complex components that non-viral ...

Artistic rendering of gene editing reagents — mRNA (red) and DNA (green and yellow) constructs — being packaged into a lipid nanoparticle (blue). UCLA researchers have developed a lipid ...