Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years.

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第一作者： Susan A,Berry

第一单位： University of Minnesota, Minneapolis, MN, USA.

作者： Susan A,Berry ^[1] ; Nicola,Longo ^[2] ; George A,Diaz ^[3] ; Shawn E,McCandless ^[4] ; Wendy E,Smith ^[5] ; Cary O,Harding ^[6] ; Roberto,Zori ^[7] ; Can,Ficicioglu ^[8] ; Uta,Lichter-Konecki ^[9] ; Beth,Robinson ^[10] ; Jerry,Vockley ^[11]

作者单位： University of Minnesota, Minneapolis, MN, USA. ^[1] University of Utah, Salt Lake City, UT, USA. ^[2] Icahn School of Medicine at Mount Sinai, New York, NY, USA. ^[3] Center for Human Genetics, University Hospitals Cleveland Medical Center and Case Western Reserve University, Cleveland, OH, USA. ^[4] Maine Medical Center, Portland, ME, USA. ^[5] Oregon Health & Science University, Portland, OR, USA. ^[6] University of Florida, Gainesville, FL, USA. ^[7] Children's Hospital of Philadelphia, Philadelphia, PA, USA. ^[8] Children's Hospital of Pittsburgh, Pittsburgh, PA, USA. ^[9] Horizon Pharma USA, Inc., Lake Forest, IL, USA. Electronic address: brobinson@horizonpharma.com. ^[10] Children's Hospital of Pittsburgh, Pittsburgh, PA, USA; University of Pittsburgh School of Medicine, Pittsburgh, PA, USA. ^[11]

关键词 Ammonia Children Glutamine Glycerol phenylbutyrate Infants Urea cycle disorders