Patients with rare diseases offer a unique perspective about their condition and their experience is important to understand to help advance therapies for rare diseases. The stories below were captured to tell their stories and help the rare disease community understand more about the patient experience. These stories are critical to inform medical product development and regulation.

FDA experts also discuss their work on rare diseases and the widespread efforts across the FDA to promote safe, effective and innovative medical products for patients.

Population estimates may change over time and there are different definitions of rare diseases.  See Developing Products for Rare Diseases and Conditions for more information. The personal opinions and views expressed in these videos do not reflect FDA's official position. 

• Stories from the Rare Disease Community 
• Rare Disease Work Across the FDA

Stories from the Rare Disease Community