The Welsh government and Vertex Pharmaceuticals have reached an agreement that will allow access to the company’s Kaftrio (ivacaftor/…
Articles by Patricia Inácio, PhD
Patient dosing has begun in a Phase 1b trial evaluating Calithera Bioscience’s oral therapy CB-280 for cystic fibrosis (CF) patients…
Orkambi (ivacaftor/lumacaftor) can significantly reduce the number of days people with cystic fibrosis (CF) require intravenous antibiotics…
Exposure to second-hand smoke in infants and children with cystic fibrosis (CF) is linked to changes in levels of…
The antibiotic-resistant bacterium Stenotrophomonas maltophilia, a dangerous bacteria for people with lung diseases such as cystic fibrosis (CF), is…
Arrowhead Pharmaceuticals has filed for approval to launch a Phase 1/2a clinical trial in New Zealand testing its candidate inhaled…
ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA…
A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) —…