Key Takeaways An experimental genetic therapy can cure most cases of the 'Bubble Boy' disease, ADA-SCIDIn the rare disorder, ...

Doses of the COVID-19 vaccine purchased through the federally-funded Vaccines for Children program only started arriving this ...

Young, now 28, was born with what’s known as osteogenesis imperfecta, or brittle bone disease. Just as the names suggests, it ...

Eliminating coverage for 33 million residents doesn't cure their ills. They can either be managed inexpensively in doctors' ...

Eliminating coverage for 33 million residents doesn't cure their ills. They can either be managed inexpensively in doctors’ ...

As more people start to opt for genetic carrier testing ahead of conception, doctors and parents-to-be are facing difficult ...

Baby KJ's medical team intend to ask the FDA to start a clinical trial that would be open to patients with seven different ...

Gene therapy is linked to better nonmotor outcomes in spinal muscular atrophy type 1 compared to first-line nusinersen, new ...

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using ‘longevity genes’ found in people who ...

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

West Virginia is at a legal and moral crossroads on childhood immunization, and Governor Patrick Morrisey put us here. Morrisey’s executive order upon taking office earlier this year said the state’s ...

A 9-year-old girl walked slowly down the clinic hallway. 10 months earlier, her days had been dominated by seizures—sudden ...