Lichter-Konecki, Uta
Sanz, Jacqueline H.
Ah Mew, Nicholas
Baumgartner, Matthias R.
Bedoyan, Jirair K.
Berry, Gerard
Berry, Susan A.
Burgard, Peter
Burrage, Lindsay
Coughlin, Curtis
Diaz, George A.
Enns, Gregory
Gallagher, Renata C.
Gropman, Andrea
Harding, Cary O.
Hoffmann, Georg F.
Le Mons, Cynthia
McCandless, Shawn E.
Merritt, J. Lawrence II
Nagamani, Sandesh C. S.
Schulze, Andreas
Seminara, Jennifer
Stricker, Tamar
Waisbren, Susan
Wong, Derek
Yudkoff, Marc
McCarter, Robert
,
Article History
Received: 12 September 2022
Revised: 29 May 2023
Accepted: 15 June 2023
First Online: 15 July 2023
Competing interests
: U.L.-K., J.H.S., and R.C. have no potential conflicts of interest regarding this study. Of the consortium authors: Mathias Baumgartner received a research fund from Nutricia and is a member of the clinical advisory boards of Hemoshear and Moderna neither of these involvements is regarding Urea Cycle disorders. George Diaz is now working for iECure. His involvement in the UCDC occurred, while he was employed by Icahn School of Medicine at Mount Sinai and he declared no competing interests regarding this manuscript. Gregory Enns receives compensation as a consultant for AllStripes, Hemoshear, Horizon Therapeutics, M6P Therapeutics, and Ultragenyx Pharmaceutical and clinical trial support from Aeglea Biotherapeutics. J Lawrence Merritt currently reports employment by, and stock ownership in, Ultragenyx Pharmaceutical Inc. His involvement in the UCDC occurred, while JLM was employed by Seattle Children’s Hospital, prior to this Ultragenyx employment and stock ownership. Andreas Schulze is consulting or on the advisory board of Ultragenyx and Horizon and conducts industry initiated clinical studies for Aeglea. Susan Waisbren consults Ultragenyx Gene Therapy.
: The Longitudinal Study of the UCDC was approved by the IRBs at all participating sites and informed consent/assent was obtained from all study participants or legal guardians.