A newly identified subtype of Castleman disease will help diagnose and properly treat thousands of patients who have been caught between existing classification systems, marking the first major ...

Gary Ablett Jr and his wife Jordan have opened up for the first time about the rare degenerative disorder that their six-year-old son Levi is currently dying of.

Menkes is a rare recessive disorder which affects copper metabolism, leading to neurodegeneration and short life expectancy.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization ...

From high blood pressure to heart disease and clinical trials, this conversation pulls back the curtain on issues that impact millions.

I am excited to update you on our overall strategy, progress, and key priorities and goals for 2025. Strategic Overview and ...

The bright and playful boy was diagnosed with a disease that accounts for just three per cent of childhood cancers.

A pharma company raised the price for a rare disease drug by 150%, arguing a new U.S. law left it no other choice.

Congress cannot discover treatments and cures for diseases, but it can support those who do. In Washington, I co-chair the Rare Disease Congressional Caucus. In this group, my colleagues and ...

Congress cannot discover treatments and cures for diseases, but it can support those who do. In Washington, I co-chair the ...

Stanford Medicine researchers have developed a blood test capable of detecting cancers, the ways cancer resists treatments ...

At just 11-years-old, Julianna Reilly has already undergone heart surgeries and wears a monitor daily to track symptoms of a ...