Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions i

This User’s Guide is intended to support the design, implementation, analysis, interpretation, and quality evaluation of registries created to increase unders

This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital provide

Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively a