Total Revenue of $560 million, exceeding guidanceCrysvita® revenue of $410 million and Dojolvi® revenue of $88 million2025 Financial ...

The biggest cash prize in TV history was given out in the February 13 finale. Find out why MrBeast is '100 percent' confident ...

Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and ...

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

The Rice lab of bioengineer Gang Bao and collaborators at Baylor College of Medicine have developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the ...

Rocket Pharmaceuticals, Inc.'s innovative therapies show promise after FDA setbacks. With potential $1B+ markets, RCKT offers ...

Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...

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

A groundbreaking study reveals how a rare combination of genetics, proteomics, and environmental factors may hold the key to ...

Explore the potential of monoclonal antibodies in Alzheimer’s treatment, including breakthroughs and challenges in this ...