A rare disease is defined as a disease that affects less than 200,000 people in the United States. Although rare diseases can oftentimes be overlooked in clinical advancement, it is estimated that such advancements would benefit over 300 million individuals worldwide. While there have been significant progress in the diagnosis and treatment of rare diseases over the last several decades, there is still a great unmet need for approaching the management of these less common diseases. In order to address this need, it will be vital to understand the challenges of managing rare diseases and how innovative approaches can enhance patient therapies.

This white paper will cover:

• Overview of rare diseases
• Traditional approaches to rare disease management
• Innovative advances in rare disease treatment