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Natural History Studies for Rare Diseases

The purpose of the video is to inform patients, patient advocates, caregivers, scientists and others who have an interest in rare diseases about the importance of natural history studies. The Food and Drug Administration’s (FDA) Office of Orphan Products Development is introducing a new grants program to support natural history studies This video is a moderated discussion between FDA physicians, a patient, a caregiver and the chair of the scientific advisory board for a patient advocacy group.