title: Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium.
abstract: The Urea Cycle Disorders Consortium (UCDC) was created as part of a larger network established by the National Institutes of Health to study rare diseases. This paper reviews the UCDC's accomplishments over the first 6years, including how the Consortium was developed and organized, clinical research studies initiated, and the importance of creating partnerships with patient advocacy groups, philanthropic foundations and biotech and pharmaceutical companies.
authors.affiliation: Children's National Medical Center, Washington, DC 20010, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Seminara
authors.forename: Mendel
authors.initials: M
authors.lastname: Tuchman
authors.forename: Lauren
authors.initials: L
authors.lastname: Krivitzky
authors.forename: Jeffrey
authors.initials: J
authors.lastname: Krischer
authors.forename: Hye-Seung
authors.initials: HS
authors.lastname: Lee
authors.forename: Cynthia
authors.initials: C
authors.lastname: Lemons
authors.forename: Matthias
authors.initials: M
authors.lastname: Baumgartner
authors.forename: Stephen
authors.initials: S
authors.lastname: Cederbaum
authors.forename: George A
authors.initials: GA
authors.lastname: Diaz
authors.forename: Annette
authors.initials: A
authors.lastname: Feigenbaum
authors.forename: Renata C
authors.initials: RC
authors.lastname: Gallagher
authors.forename: Cary O
authors.initials: CO
authors.lastname: Harding
authors.forename: Douglas S
authors.initials: DS
authors.lastname: Kerr
authors.forename: Brendan
authors.initials: B
authors.lastname: Lanpher
authors.forename: Brendan
authors.initials: B
authors.lastname: Lee
authors.forename: Uta
authors.initials: U
authors.lastname: Lichter-Konecki
authors.forename: Shawn E
authors.initials: SE
authors.lastname: McCandless
authors.forename: J Lawrence
authors.initials: JL
authors.lastname: Merritt
authors.forename: Mary Lou
authors.initials: ML
authors.lastname: Oster-Granite
authors.forename: Margretta R
authors.initials: MR
authors.lastname: Seashore
authors.forename: Tamar
authors.initials: T
authors.lastname: Stricker
authors.forename: Marshall
authors.initials: M
authors.lastname: Summar
authors.forename: Susan
authors.initials: S
authors.lastname: Waisbren
authors.forename: Marc
authors.initials: M
authors.lastname: Yudkoff
authors.forename: Mark L
authors.initials: ML
authors.lastname: Batshaw
mesh_terms: D003299:Cooperative Behavior; D006801:Humans; D007373:Interinstitutional Relations; D008137:Longitudinal Studies; D035583:Rare Diseases / Q000628:therapy*; D012042:Registries; D056806:Urea Cycle Disorders, Inborn / Q000628:therapy*