Scientists have long believed that disease-causing genetic variants inevitably lead to disease. But a sea change of research ...

In February, 2025, we reported in The Lancet the human toll of the UK's prolonged refusal to introduce newborn screening for spinal muscular atrophy, despite overwhelming international evidence ...

There may be no worse place to be than in a body that’s failing when the doctors can’t tell you what’s wrong. Slipping ...

Lisa Hayden-Johnson subjected her son, Matthew, to needless medical procedures - now he and his sister have spoken out ...

Kristin Entler‘s debut poetry collection, Of Water Never Ceasing, documents the struggles of living with chronic illness. Drawing from Entler’s own experiences of living with cystic fibrosis, Of Water ...

A Houston woman was 72 years old when she learned she'd had a rare disorder her entire life. How was that possible?

Bronchiectasis associated with cystic fibrosis (CF) is well characterized, but the role of partial CFTR dysfunction in patients with non-CF bronchiectasis remains less clear. Patients with ...

Insmed is joining forces with an organisation representing people living with the chronic lung disorder bronchiectasis to carry out a large-scale, real-world study of its recently approved therapy ...

Prime Medicine possesses promising gene editing technology but faces significant dilution risk and cash constraints. Read ...

Sionna Therapeutics (NASDAQ:SION) said its PreciSION CF Phase 2a proof-of-concept study of SION-719 added to Trikafta did not ...

A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta. Shares ...