Conducting an investigator-initiated randomized double-blinded intervention trial in acute decompensation of inborn errors of metabolism: Lessons from the N-Carbamylglutamate Consortium.

Ah Mew, Nicholas; Cnaan, Avital; McCarter, Robert; Choi, Henry; Glass, Penny; Rice, Katie; Scavo, Louis; Gillespie, Catherine W; Diaz, George A; Berry, Gerard T; Wong, Derek; Konczal, Laura; McCandless, Shawn E; Coughlin Ii, Curtis R; Weisfeld-Adams, James D; Ficicioglu, Can; Yudkoff, Mark; Enns, Gregory M; Lichter-Konecki, Uta; Gallagher, Renata; Tuchman, Mendel

Ah Mew, Nicholas; Cnaan, Avital; McCarter, Robert; Choi, Henry; Glass, Penny; Rice, Katie; Scavo, Louis; Gillespie, Catherine W; Diaz, George A; Berry, Gerard T; Wong, Derek; Konczal, Laura; McCandless, Shawn E; Coughlin Ii, Curtis R; Weisfeld-Adams, James D; Ficicioglu, Can; Yudkoff, Mark; Enns, Gregory M; Lichter-Konecki, Uta; Gallagher, Renata; Tuchman, Mendel.

Ah Mew N; Children's National Health System, Washington, DC, USA.
Cnaan A; Children's National Health System, Washington, DC, USA.
McCarter R; Children's National Health System, Washington, DC, USA.
Choi H; Children's National Health System, Washington, DC, USA.
Glass P; Children's National Health System, Washington, DC, USA.
Rice K; Children's National Health System, Washington, DC, USA.
Scavo L; Children's National Health System, Washington, DC, USA.
Gillespie CW; Children's National Health System, Washington, DC, USA.
Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Berry GT; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Wong D; UCLA Mattel Children's Hospital, Los Angeles, CA, USA.
Konczal L; Rainbow Babies and Children's Hospital, Cleveland, OH, USA.
McCandless SE; Children's Hospital Colorado and University of Colorado Denver, Aurora, CO, USA.
Coughlin Ii CR; Children's Hospital Colorado and University of Colorado Denver, Aurora, CO, USA.
Weisfeld-Adams JD; Children's Hospital Colorado and University of Colorado Denver, Aurora, CO, USA.
Ficicioglu C; Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Yudkoff M; Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Enns GM; Lucile Packard Children's Hospital Stanford, Palo Alto, CA, USA.
Lichter-Konecki U; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Gallagher R; UCSF Benioff Children's Hospital, San Francisco, CA, USA.
Tuchman M; Children's National Health System, Washington, DC, USA.

Transl Sci Rare Dis ; 3(3-4): 157-170, 2018 Dec 20.

Article en En | MEDLINE | ID: mdl-30613471

Palabras clave

Rare diseases; clinical trials; hyperammonemia; inborn errors of metabolism; methylmalonic acidemia; organic acidemia; propionic acidemia; urea cycle disorders

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