Learning that your child has neurofibromatosis type 1 (NF1) comes with a range of emotions and practical concerns. While many people diagnosed with NF1 will have a normal life expectancy, the ...

CLARYVILLE, N.Y. (AP) — Autoimmune diseases like lupus, myositis and forms of arthritis can strike children, too. At a sleepaway camp in upstate New York, some young patients got a chance to just be ...

For too long, the promise of personalized therapies has been tantalizingly close, yet frustratingly out of reach for rare-disease families.

A doctor advising … sleepaway camp? That’s how a 12-year-old diagnosed with lupus found himself laughing on a high-ropes course as fellow campers hoisted him into the air. Six federal scientists run ...

All UK children could be offered screening for type 1 diabetes using a simple finger-prick blood test, say researchers who have been running a large study. Currently, many young people go undiagnosed ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...