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Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years.
Berry, Susan A; Longo, Nicola; Diaz, George A; McCandless, Shawn E; Smith, Wendy E; Harding, Cary O; Zori, Roberto; Ficicioglu, Can; Lichter-Konecki, Uta; Robinson, Beth; Vockley, Jerry.
Affiliation
  • Berry SA; University of Minnesota, Minneapolis, MN, USA.
  • Longo N; University of Utah, Salt Lake City, UT, USA.
  • Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • McCandless SE; Center for Human Genetics, University Hospitals Cleveland Medical Center and Case Western Reserve University, Cleveland, OH, USA.
  • Smith WE; Maine Medical Center, Portland, ME, USA.
  • Harding CO; Oregon Health & Science University, Portland, OR, USA.
  • Zori R; University of Florida, Gainesville, FL, USA.
  • Ficicioglu C; Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Lichter-Konecki U; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
  • Robinson B; Horizon Pharma USA, Inc., Lake Forest, IL, USA. Electronic address: brobinson@horizonpharma.com.
  • Vockley J; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA; University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
Mol Genet Metab ; 122(3): 46-53, 2017 11.
Article in En | MEDLINE | ID: mdl-28916119

Full text: 1 Database: MEDLINE Main subject: Phenylbutyrates / Urea Cycle Disorders, Inborn / Glutamine / Glycerol / Ammonia Type of study: Observational_studies / Risk_factors_studies Language: En Journal: Mol Genet Metab Year: 2017 Type: Article Affiliation country: United States

Full text: 1 Database: MEDLINE Main subject: Phenylbutyrates / Urea Cycle Disorders, Inborn / Glutamine / Glycerol / Ammonia Type of study: Observational_studies / Risk_factors_studies Language: En Journal: Mol Genet Metab Year: 2017 Type: Article Affiliation country: United States