A provision protecting expensive medications, used to treat multiple rare diseases, from the Medicare drug price negotiations ...

For Bethany Duckworth, being an active mom wasn’t just a dream, it was her plan. "It's depressing," Duckworth said. "It's sad ...

June is Alzheimer's and Brain Awareness Month. Colbie Schueneman, 6, is sharing her story with KIF1A, also known as KAND.

GRIN-related neurodevelopmental disorder (GRIN-NDD) is a family of rare, genetically defined neurodevelopmental disorders ...

Researchers at the Gray Faculty of Medical and Health Sciences at Tel Aviv University have developed a model that accurately ...

Three major Mount Sinai research studies offer new hope for patients facing chronic lymphocytic leukemia (CLL), a rare type ...

The Orphan Cures Act has been added back into the Senate version of President Donald Trump’s “big, beautiful bill” after being ...

A yearslong effort to challenge Michigan’s practice of storing millions of dried blood samples from newborn babies has been ...

A massive analysis recently published in the journal Aging Cell revealed that rapamycin — the immunosuppressant drug turned ...

Critically ill children and their families are given life-changing wishes by Make-A-Wish, often involving Disney. Disney is ...

For decades, international researchers have received a small slice of the National Institutes of Health budget. In 2024, out ...

Hansen's Disease, more commonly known as leprosy, is a chronic disease that can lead to physical impairment. Today it exists ...