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…