A new inhalable gene therapy for cystic fibrosis (CF) has entered Phase I clinical trials, a major milestone on the journey ...

Once-daily vanzacaftor-tezacaftor-deutivacaftor was safe and well-tolerated in children with cystic fibrosis, while ...

From life-altering genetic conditions to promising new treatments, here's what you need to know about 7 inherited diseases ...

Background Early-life inflammation has long been recognised as a key pathophysiological process in the evolution of cystic ...

Around 75% of rare diseases are diagnosed in childhood, with most before the age of two. With 30% of rare disease patients ...

Having a child with vs. without cystic fibrosis heightened the odds for job lock and curtailed employment among parents/caregivers, according to a research letter published in JAMA Pediatrics.“U.S.

Tauranga boy Riaan Hamilton has cystic fibrosis – the 3-year-old often gets sick, has been hospitalised several times, and is ...

Findings from a new Dartmouth-led study, published in the journal mBio, highlight key differences in the gut microbiome ...

Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.

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 ...