Feb 9 (Reuters) - Regenxbio said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for ...

Among them was David Liu, a biochemist at the Broad Institute and Harvard University in Cambridge, Massachusetts. Liu isn’t a ...

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 ...

The author's son has hemophilia, a rare disorder requiring expensive, life-saving medication. The TrumpRx program aims to ...

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 ...

Many people may have a dim view of their fat tissue, yet scientists have come to recognize adipose as a necessary and ...

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...

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

Philanthropic funding from the FA Alliance Innovation Fund awarded to five Oxford researchers to support development of novel therapeutics ...

"The situation is under constant monitoring and all necessary public health measures are in place," the Indian Health ...