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UX007 for the treatment of long chain-fatty acid oxidation disorders: Safety and efficacy in children and adults following 24weeks of treatment.
Vockley, J; Burton, B; Berry, G T; Longo, N; Phillips, J; Sanchez-Valle, A; Tanpaiboon, P; Grunewald, S; Murphy, E; Humphrey, R; Mayhew, J; Bowden, A; Zhang, L; Cataldo, J; Marsden, D L; Kakkis, E.
Afiliación
  • Vockley J; University of Pittsburgh, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA, USA. Electronic address: gerard.vockley@chp.edu.
  • Burton B; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA.
  • Berry GT; Boston Children's Hospital, Boston, MA, USA.
  • Longo N; University of Utah, Salt Lake City, UT, USA.
  • Phillips J; Vanderbilt University Medical Center, Nashville, TN, USA.
  • Sanchez-Valle A; University of South Florida, Morsani College of Medicine, Tampa, FL, USA.
  • Tanpaiboon P; Children's National Medical Center, Washington, DC, USA.
  • Grunewald S; Great Ormond Street Hospital, UCL Institute of Child Health, London, UK.
  • Murphy E; Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
  • Humphrey R; University of Montana, Missoula, MT, USA.
  • Mayhew J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Bowden A; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Zhang L; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Cataldo J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Marsden DL; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
  • Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, CA, USA.
Mol Genet Metab ; 120(4): 370-377, 2017 04.
Article en En | MEDLINE | ID: mdl-28189603
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Resistencia Física / Triglicéridos / Ácidos Grasos / Errores Innatos del Metabolismo Lipídico Tipo de estudio: Clinical_trials / Observational_studies / Risk_factors_studies Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2017 Tipo del documento: Article
Texto completo
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Resistencia Física / Triglicéridos / Ácidos Grasos / Errores Innatos del Metabolismo Lipídico Tipo de estudio: Clinical_trials / Observational_studies / Risk_factors_studies Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2017 Tipo del documento: Article