NORD Guide to Rare Disorders is a comprehensive, practical, authoritative guide to the diagnosis and management of more than 800 rare diseases. The diseases are

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