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Long-term safety and efficacy of glycerol phenylbutyrate for the management of urea cycle disorder patients.
Diaz, George A; Schulze, Andreas; Longo, Nicola; Rhead, William; Feigenbaum, Annette; Wong, Derek; Merritt, J Lawrence; Berquist, William; Gallagher, Renata C; Bartholomew, Dennis; McCandless, Shawn E; Smith, Wendy E; Harding, Cary O; Zori, Roberto; Lichter-Konecki, Uta; Vockley, Jerry; Canavan, Colleen; Vescio, Thomas; Holt, Robert J; Berry, Susan A.
Afiliación
  • Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Schulze A; University of Toronto and The Hospital for Sick Children, Toronto, ON, Canada.
  • Longo N; University of Utah, Salt Lake City, UT, USA.
  • Rhead W; Medical College of Wisconsin, Milwaukee, WI, USA.
  • Feigenbaum A; University of Toronto and The Hospital for Sick Children, Toronto, ON, Canada.
  • Wong D; David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.
  • Merritt JL; Seattle Children's Hospital/University of Washington, Seattle, WA, USA.
  • Berquist W; Stanford University Medical Center & Lucile Packard Children's Hospital, Stanford, CA, USA.
  • Gallagher RC; University of California, San Francisco, CA, USA.
  • Bartholomew D; Ohio State University and Nationwide Children's Hospital, Columbus, OH, USA.
  • McCandless SE; Children's Hospital Colorado and University of Colorado Denver, Aurora, CO, 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.
  • Lichter-Konecki U; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
  • Vockley J; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA; University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
  • Canavan C; Horizon Therapeutics plc, Lake Forest, IL, USA.
  • Vescio T; Horizon Therapeutics plc, Lake Forest, IL, USA.
  • Holt RJ; Horizon Therapeutics plc, Lake Forest, IL, USA. Electronic address: rholt@horizontherapeutics.com.
  • Berry SA; University of Minnesota, Minneapolis, MN, USA.
Mol Genet Metab ; 127(4): 336-345, 2019 08.
Article en En | MEDLINE | ID: mdl-31326288

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenilbutiratos / Trastornos Innatos del Ciclo de la Urea / Glicerol Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged País/Región como asunto: America do norte Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Fenilbutiratos / Trastornos Innatos del Ciclo de la Urea / Glicerol Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged País/Región como asunto: America do norte Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos