Insmed’s ‘ginormous’ lung drug sales; J&J’s US pricing deal
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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,…
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…