Creutzfeldt-Jakob disease is an extremely rare and fatal brain-wasting disease that's like a human version of "mad cow." ...

We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...

The event featured not only Austin Laurette, who is "the unbreakable boy," but also some of the stars of the movie, Zachary ...

Its architect's daughter has cystic fibrosis—and benefits from a "miracle drug" backed by an agency he's attacking.

In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...

Five years ago, the COVID-19 pandemic served as a wake-up call. Since then, health experts worldwide have sat on tenterhooks, ...

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

To know Bentley Shelton is to love him. “He brings light to everybody that meets him,” said Brittany Shelton, Bentley’s mom. “He always ...

Tuesday, the agency signed off on SpringWorks’ Gomekli (mirdametinib) as a treatment for the rare genetic disorder ...

A surprising new study suggests that COVID-19 may not have originated from bats or pangolins, but rather from a rare fusion of human diseases. Using an advanced AI-driven approach called max-logistic ...

TEXARKANA, Texas - A Texarkana, Texas boy battling a rare genetic disease is preparing to head to Arkansas Children's ...

Mereo BioPharma shifts focus to rare diseases with setrusumab showing strong results for Osteogenesis Imperfecta. Learn more on MREO stock here.