Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only ...

Eric Dane died after battling ALS. Here’s what to know about ALS, the progressive neurological disease that affects nerve ...

Globally, an estimated 300 million people live with a rare disease. They typically experience isolation, diagnostic difficulties, lack of therapies, fragmented care, stigma, a struggle for recognition ...

Scientists have identified how specific genetic changes function in cells to influence disease risk and other human health ...

While dementia and Alzheimer’s disease are terms that are often used interchangeably, they don’t mean the same thing. Understanding the difference isn’t just helpful for clarity, it’s essential for ...

Rare disease researchers have long known that viewing each patient as a source of insight rather than a single data point is critical to developing the most effective treatments.

Nine out of 10 people with chronic kidney disease only find out when it progresses to later stages. Here’s what we know about its risk factors—and why there's new reason to hope for more effective ...