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        Medical products for rare diseases and conditions

Personal Stories About Rare Diseases Home
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          Medical products for rare diseases and conditions
          Personal Stories About Rare DiseasesMedical products for rare diseases and conditions Personal Stories About Rare Diseases
  	
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    PrintPatients 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 FDAStories from the Rare Disease Community

**Braulio Hernandez** - Hodgkin’s Lymphoma
			**Lauren Edwards** - Gaucher Disease
			**Shawn and Harper** - SCN2A-related disorderSee more stories from the rare disease community.

Rare Disease Work Across the FDA

**Aaron Friedman -**Regulatory Counsel for the Office of Orphan Products Development
			**Julienne Vaillancourt -**Rare Disease Liaison for the Center for Biologics Evaluation and Research
			**Kerry Jo Lee-**Acting Associate Director for Rare Diseases for Center for Drugs Evaluation & ResearchSee more stories about FDA's rare disease work.

                                                                                  Content current as of:
      03/18/2022Personal Stories About Rare Diseases
 
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