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Glycerol phenylbutyrate efficacy and safety from an open label study in pediatric patients under 2 months of age with urea cycle disorders.
Longo, Nicola; Diaz, George A; Lichter-Konecki, Uta; Schulze, Andreas; Inbar-Feigenberg, Michal; Conway, Robert L; Bannick, Allison A; McCandless, Shawn E; Zori, Roberto; Hainline, Bryan; Ah Mew, Nicholas; Canavan, Colleen; Vescio, Thomas; Kok, Teresa; Porter, Marty H; Berry, Susan A.
  • Longo N; University of Utah, Salt Lake City, UT, USA.
  • Diaz GA; Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Lichter-Konecki U; Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
  • Schulze A; Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
  • Inbar-Feigenberg M; Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
  • Conway RL; Wayne State University School of Medicine, Detroit, MI, USA.
  • Bannick AA; Wayne State University School of Medicine, Detroit, MI, USA.
  • McCandless SE; University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Aurora, CO, USA.
  • Zori R; University of Florida, Gainesville, FL, USA.
  • Hainline B; Indiana University School of Medicine, Indianapolis, IN, USA.
  • Ah Mew N; Children's National Medical Center, Washington, DC, USA.
  • Canavan C; Horizon Therapeutics plc, Deerfield, IL, USA.
  • Vescio T; Horizon Therapeutics plc, Deerfield, IL, USA.
  • Kok T; Horizon Therapeutics plc, Deerfield, IL, USA. Electronic address: Tkok@horizontherapeutics.com.
  • Porter MH; Horizon Therapeutics plc, Deerfield, IL, USA.
  • Berry SA; University of Minnesota, Minneapolis, MN, USA.
Mol Genet Metab ; 132(1): 19-26, 2021 01.
Article en En | MEDLINE | ID: mdl-33388234
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fenilbutiratos / Hiperamonemia / Trastornos Innatos del Ciclo de la Urea / Glicerol Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
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PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Fenilbutiratos / Hiperamonemia / Trastornos Innatos del Ciclo de la Urea / Glicerol Límite: Child, preschool / Female / Humans / Infant / Male / Newborn Idioma: En Año: 2021 Tipo del documento: Article