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 ...

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 ...

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

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