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 ...
Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like ...
Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.
Amgen has denied a request by the FDA to voluntarily withdraw its rare disease drug Tavneos from the market, the company said ...
5don MSN
This 11-year-old has battled a rare disease for years — a breakthrough drug helped deliver relief
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological ...
GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...
Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...
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 ...
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 ...
Natalie Banner, Chief Ethics and Engagement Officer for Genomics England, shares how we can help to address inequities in ...
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