Rare Diseases

Florida Institute for Pediatric Rare Diseases and Representative Adam Anderson ushering in a new era of preventative care…

Isla Steed is your fairly typical teenager, according to her mum. The 14-year-old is “a stubborn little person”…

SINGAPORE – Not all medicines work the same way on every patient, and part of the answer lies…

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…

The standard treatment is a liver transplant, but this entails a high risk of complications in infants. Thanks…

For the first time, scientists say they have reached into the genome of a severely ill child and…

For European drugmakers there are still many unknowns about how the MFN scheme — and overall tariffs on…

image: ©Rost-9D | iStock Researchers using patient-derived stem cells reveal a rare ALS mutation triggers a chronic stress…

GeneDx to Partner with Florida’s Sunshine Genetics Network, Launching Nation’s First State-Backed Genomic Newborn Screening Program