While the drop in Ultragenyx stock certainly provides a lower entry point for new investors, the more prudent choice right ...

Industry data shows small molecules achieve 5–10% approval rates over 15–20 years; antisense oligonucleotides (“ASOs”) are shifting the odds through rational design. Six new antisense drugs gained FDA ...

Final results from the VALOR trial and its open-label extension demonstrated the clinical benefit of tofersen in adults with ...

As of Tuesday, December 30, Ultragenyx Pharmaceutical Inc.’s RARE share price has surged by 9.99%, which has investors questioning if this is right time to sell.

Sean Ryder, professor at UMass Chan Medical School, has written a book to delve the mysteries of ribonucleic acid as a tool to fight diseases. Messenger ribonucleic acid, or mRNA, is in the public ...

Historically, people with amyotrophic lateral sclerosis (ALS) experience a relentless decline in neurological function that ...

By broadening definition of success to include sustained low-level HBsAg with suppressed HBV DNA, we can recognize clinically ...

Ultragenyx Pharmaceutical (RARE) remains a Sell after its Phase 3 OI program failed to meet primary fracture reduction ...

Sapu BioScience today announced the publication of a new peer-reviewed study in the International Journal of Molecular Sciences that strengthens the scientific foundation for biomarker-guided taxane ...

VESALIUS-CV was big in 2025, but studies looking at triglycerides, CRISPR, and oral GLP-1s also made the highlight reel.

However, significant changes have been underway, converging regulatory breakthroughs, maturing AI capabilities, and a broader ...