Rare disease clinical trials face a confluence of challenges: limited patient populations, heterogeneity in disease progression, and often a lack of established outc...

Who better to shape clinical research in rare disease than those living with the conditions. Patient and public involvement in clinical research is crucial for ensur...

Rare diseases, which affect >30 million individuals in the United States (US), have historically taken a back seat to more common diseases such as cancer, diabete...

Mislabelled vials or mishandled specimens in clinical trials can compromise data accuracy, potentially affecting patient safety and outcomes. With increasing study s...