The EveryLife Foundation for Rare Diseases (EveryLife Foundation) announced the awardees for the 2025 RareVoice Awards, which ...
U.S. President Donald Trump signed a spending package into law Tuesday that reauthorizes the FDA’s previously stalled rare pediatric disease priority review voucher program, among other initiatives, ...
Alström syndrome affects roughly one in 500,000 people. In May, Fort Worth will host a symposium of researchers looking into treatments.
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding ...
This Rare Disease Day, observed on Feb. 28 worldwide, the American Kidney Fund is committed to improving the understanding of ...
4don 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 ...
Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic defect.
GeneDx, a fast-growing biotech doing exome and genomic testing, has seen big returns on Wall Street as appetite for its tests ...
For Aaron and Krystle Myers, the signs that something wasn’t right with their daughter Amelia didn’t come all at once.
Hackathons using AlphaGenome and other AI models are hunting down the genetic causes of devastating conditions that have ...
Highlights advancing clinical-stage pipeline, including two major value-creating programs for primary immunodeficiencies ...
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 ...
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