Establishing a consortium for the study of rare diseases: The Urea Cycle Disorders Consortium.
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.
Seminara
Jennifer
J
Children's National Medical Center, Washington, DC 20010, USA.
Tuchman
Mendel
M
Krivitzky
Lauren
L
Krischer
Jeffrey
J
Lee
Hye-Seung
HS
Lemons
Cynthia
C
Baumgartner
Matthias
M
Cederbaum
Stephen
S
Diaz
George A
GA
Feigenbaum
Annette
A
Gallagher
Renata C
RC
Harding
Cary O
CO
Kerr
Douglas S
DS
Lanpher
Brendan
B
Lee
Brendan
B
Lichter-Konecki
Uta
U
McCandless
Shawn E
SE
Merritt
J Lawrence
JL
Oster-Granite
Mary Lou
ML
Seashore
Margretta R
MR
Stricker
Tamar
T
Summar
Marshall
M
Waisbren
Susan
S
Yudkoff
Marc
M
Batshaw
Mark L
ML
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*