Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

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 ...

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 ...

An experimental treatment reduces seizures and other symptoms in children with a type of epilepsy called Dravet syndrome.

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 ...

UNICEF’s work to improve care for children living with NCDs in Zimbabwe helped Kelly, 10, receive diabetes diagnosis and treatment. Clinical matron Mary Thema tests 10-year-old Kelly's blood glucose ...

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...