Former Wallaby Nathan Charles fights motor neurone disease
Former Wallaby Nathan Charles has been diagnosed with motor neurone disease. Sydney-born Charles, 37, represented the Wallabies in…
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.…
‘I never thought I’d make 30 or get married but now I am thanks to wonder drug’
Hailey Mann, who has cystic fibrosis, was preparing for the end of her life at 24 when the…
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…
BBC breakfast hosts deliver heartbreaking news after star’s ‘life-limiting’ diagnosis
BBC Breakfast’s Naga Munchetty and Charlie Stayt delivered the news on Saturday. BBC Breakfast hosts Naga Munchetty and…
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…