Scientists design new RNA therapy to bypass mutations behind thousands of genetic diseases
Scientists engineered transfer RNA to bypass premature genetic stop signals and restore production of full-length proteins in cystic…
Wearable Sweat Test Stickers for Cystic Fibrosis
Research indicates that flexible stickers can accurately measure sweat chloride levels in cystic fibrosis patients in under an…
Using DNA sequencing for accurate identification of microbial infections in cystic fibrosis patients
Lung infections are a constant risk for people living with cystic fibrosis (CF), a life-threatening genetic disorder which…
Colorado can’t cap price of arthritis drug Enbrel, judge rules
Colorado can’t cap the price of arthritis medication Enbrel while a lawsuit challenging the state’s drug affordability board’s…
A Loophole Brings Cystic Fibrosis Patients a ‘Miracle Drug’ in Generic Form
Josua Lottering stepped out the airport doors in the Bangladesh capital, Dhaka, and doubled over in coughing spasms.…
Insmed’s ‘ginormous’ lung drug sales; J&J’s US pricing deal
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free…
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…