Agency staff "strongly recommended" a sham surgery-controlled trial be conducted before an approval filing, a stance one ...

A key advantage of using RWD in a natural history study is gaining diverse and representative data. RWD covers broad patient populations, including those with comorbidities, various age groups, and ...

“Designing treatments unique to individual patients has always been the promised goal of personalized medicine. After 25 years the FDA has, for the first time, outlined a framework to facilitate these ...

The results show that those with low vitamin D levels had a significantly higher risk of hospitalization related to respiratory tract infection compared with those whose 25 (OH)D levels were 75 nmol/L ...

TD Cowen 46th Annual Health Care Conference March 2, 2026 2:30 PM ESTCompany ParticipantsDave Gancarz - Chief Business ...

Protein S deficiency represents a rare but significant hereditary thrombophilia that poses substantial risks during pregnancy ...

New research shows that changing how lawmakers discuss mass shootings has little effect on voter beliefs. Most Americans already support various firearm restrictions, and their entrenched views do not ...

Held Type A meeting with FDA to discuss AMT-130 for Huntington’s disease; Company evaluating Phase III development considerations and plans to ...

Protein S deficiency represents a rare but significant hereditary thrombophilia that poses substantial risks during pregnancy, yet clinical management ...

A 20-year follow-up of older adults in the ACTIVE randomized trial linked to Medicare claims found that speed of processing cognitive training with booster sessions was associated with a significantly ...

PEACE-2 finds adding cabazitaxel to ADT plus radiotherapy fails to improve survival in high-risk localized prostate cancer, while increasing severe toxicities and deaths.

In today’s ACT Brief, we explore how Bayesian trial designs are gaining traction in rare disease research, what priorities clinical operations leaders should focus on in 2026, and how rare disease ...