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Natural History Studies for Rare Diseases

Published on Feb 29, 2016

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.

What is a Natural History Study?

(FDA) PDF

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Importance of Natural History Studies in Rare Diseases Anne R. Pariser, M.D. Associate Director for Rare Diseases Office of New Drugs Center for Drug Evaluation and …

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