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

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

While the leading cause of kidney disease is diabetes, many other factors can lead to kidney disease and failure – including ...

The programme will focus on accelerating treatments for rare diseases, most of which currently lack approved therapies.

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...

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 ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Research on rare diagnoses and the development of precision medicine depend on patients being able to share their health data in a secure and ethical manner. The research study, published in ...

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

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

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 ...