At Web Summit Qatar, AI-powered biotech startups describe how automation, data, and gene editing are filling labor gaps in ...

Ever looked in the mirror and wondered what incredible, and sometimes unsettling, secrets your own body might be holding?

The Consolidated Appropriations Act of 2026, signed by Trump on Feb. 3, includes a provision reinstating the Mikaela Naylon ...

U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare ...

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...

Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...

Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...

Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.

When you have a rare disease, managing doctor's appointments, prescriptions and care plans is difficult enough — figuring out what Medicare will (and won't) cover often adds another layer of stress.

For Aaron and Krystle Myers, the signs that something wasn’t right with their daughter Amelia didn’t come all at once.

The number of Centres of Excellence under the National Policy for Rare Diseases (NPRD) has been increased from eight to 15 ...

The Rare Disease Researcher Interest Group seminar series brings together researchers from different fields to learn about and discuss rare disease research, regardless of experience level with rare d ...