In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...

Four American families aim to raise $1.15 million by October to fund a phase 3 gene therapy trial for their children with ...

Working with agency partner DCX Growth Accelerator, GeneDX takes direct aim at the issue of underdiagnosis of pediatric rare diseases.

In a study published in Nature Medicine, Leone and her team reported the first targeted gene therapy to restore myelin, the ...

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

The donation came from the David Koch Jr. Foundation to enable researchers at Stanford to redefine treatment for pediatric ...

Even though Liam Dahlberg was vaccinated before his death earlier this year, he attended school in an Indiana county where ...

A virus known as coxsackievirus A16, commonly referred to as hand, foot and mouth disease, is spreading quickly among ...

LCH is a rare disease where immune cells build up and create tumors and tissue damage. Sometimes LCH causes a skin rash or an ...

October 14, 2025 – PRESSADVANTAGE – Juvenile Idiopathic Arthritis (JIA) is the most common rheumatic disease in children, affecting thousands of young patients across the United States. For some, it ...

Over 212,000 Afghan children are now at risk of acute watery diarrhoea and other deadly waterborne diseases, according to the ...