Chronic liver disease threatens millions across Europe, report finds
Europe is facing a growing chronic liver disease threat, according to a new Series published today in The…
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…
Kiwi family move to Australia to get cystic fibrosis drug Trikafta for daughter
Adalyn Delaney was suffering the effects of CF before she was even born, with health staff discovering during…
Wegovy® approved by FDA for the treatment of adults with noncirrhotic MASH with moderate to advanced liver fibrosis
“Today’s decision by the FDA reflects the continued progress in how we understand and treat patients with MASH,…
Antimalarial drug enhances effect of aminoglycosides in genetic disorders
The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as well…