Katherine Stueland, GeneDx CEO, joins 'Squawk Box' to discuss diagnosing children with rare diseases, cost structure of the ...

From symptoms to the illness' risk factors — here’s what you need to know about the disease, which can only be detected by ...

The survey primarily focused on rare disease patients between the ages of 16 and 23, as well as caregivers between 25 to 55 years old. Hemophilia, albinism, cystic fibrosis and Gaucher disease were ...

Acute necrotizing encephalopathy is deadly in more than a quarter of kids diagnosed with the inflammatory brain disease.

Maryland-based biotech NextCure is looking for partners to advance a candidate for the currently incurable rare disease ...

The head of Italian group Recordati expects treatments for rare diseases to be exempted from the 15% tariff the United States plans to impose on imported European pharmaceutical products, he said on ...

New data from the Journal of the American Medical Association shows an unusual rise in cases of a rare and potentially deadly ...

Rising high school senior Khartik Uppalapati says he felt alienated when he was diagnosed with a rare lymphatic disorder ...

The Purdy family shares their insights on navigating their child’s rare neurodegenerative disease and their direct ...

For most Americans, the letters "NTM" don’t mean anything. But for tens of thousands of people across the country, they ...

OpEd: The Trump administration’s deep cuts to medical research at universities and the NIH ensures that those suffering with ...