July 18, 2016
Hear rare disease patients and their families share their stories in the new NCATS video series. The series aims to raise awareness of the importance of collaborative research. It features an interview with Dr. Petra Kaufmann, Director of the Office of Rare Diseases Research and Division of Clinical Innovation at NCATS, as well as with patients and families who know what it's like to live with the rare diseases MED23 gene defect, generalized lipodystrophy, relapsing polychondritis, Vici syndrome, hereditary hemorrhagic telangiectasia, epidermolysis bullosa, Phelan-McDermid syndrome, phenylketonuria, mitochondrial disease complex 3 with MELAS syndrome, and glutathione synthetase disorder.
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