Insmed’s ‘ginormous’ lung drug sales; J&J’s US pricing deal
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Enterprise Therapeutics publishes results of Phase 1 study of ETD001, a novel inhaled ENaC blocker for treatment of Cystic Fibrosis, in The Journal of Cystic Fibrosis
Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve…
Cystic fibrosis patients on triple-drug therapy can safely reduce daily lung treatments
A new multi-site study led by researchers at CU Anschutz shows that people with cystic fibrosis (CF) who…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
Genetic investigation of sinopulmonary diseases in Vietnam: seeking specific causes from non-specific symptoms | Orphanet Journal of Rare Diseases
O’Donnell AE. Bronchiectasis – a clinical review. N Engl J Med. 2022;387(6):533–45. Article PubMed Google Scholar Flume PA,…
More families raising children with cystic fibrosis staying in the bush
Connie Lacey, 4, and Sienna Machin, 10, are like sisters, but they are not allowed to be near…
Pacbio’s Puretarget simplifies carrier screening
Up to 71% of people carry at least one pathogenic variant that could contribute to development of a…
U of A to honour biology grad for breakthrough in treating cystic fibrosis
A biomedical innovator and University of Alberta graduate whose breakthrough in treating cystic fibrosis has given tens of…
Rescued by fat bubbles: Scientists treat rare genetic disease with designer molecule | Lifestyle
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with…