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Results from a 78-week, single-arm, open-label phase 2 study to evaluate UX007 in pediatric and adult patients with severe long-chain fatty acid oxidation disorders (LC-FAOD).
Vockley, Jerry; Burton, Barbara; Berry, Gerard T; Longo, Nicola; Phillips, John; Sanchez-Valle, Amarilis; Tanpaiboon, Pranoot; Grunewald, Stephanie; Murphy, Elaine; Bowden, Alexandra; Chen, Wencong; Chen, Chao-Yin; Cataldo, Jason; Marsden, Deborah; Kakkis, Emil.
Afiliação
  • Vockley J; School of Medicine and Graduate School of Public Health, University of Pittsburgh, Pittsburgh, Pennsylvania, USA.
  • Burton B; Children's Hospital of Pittsburgh, UPMC, 4401 Penn Avenue, Pittsburgh, Pennsylvania, 15224, USA.
  • Berry GT; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
  • Longo N; Boston Children's Hospital, Boston, Massachusetts, USA.
  • Phillips J; University of Utah, Salt Lake City, Utah, USA.
  • Sanchez-Valle A; Vanderbilt University Medical Center, Nashville, Tennessee, USA.
  • Tanpaiboon P; USF Health, Morsani College of Medicine, Tampa, Florida, USA.
  • Grunewald S; Children's National Health System, Washington, District of Columbia, USA.
  • Murphy E; Great Ormond Street, UCL Institute of Child Health, London, UK.
  • Bowden A; National Hospital for Neurology and Neurosurgery, London, UK.
  • Chen W; Ultragenyx Pharmaceutical Inc., Novato, California, USA.
  • Chen CY; Ultragenyx Pharmaceutical Inc., Novato, California, USA.
  • Cataldo J; Ultragenyx Pharmaceutical Inc., Novato, California, USA.
  • Marsden D; Ultragenyx Pharmaceutical Inc., Novato, California, USA.
  • Kakkis E; Ultragenyx Pharmaceutical Inc., Novato, California, USA.
J Inherit Metab Dis ; 42(1): 169-177, 2019 01.
Article em En | MEDLINE | ID: mdl-30740733
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Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 2_ODS3 / 3_ND / 4_TD / 6_ODS3_enfermedades_notrasmisibles / 7_ODS3_muertes_prevenibles_nacidos_ninos Base de dados: MEDLINE Assunto principal: Oxirredução / Triglicerídeos / Ácidos Graxos / Erros Inatos do Metabolismo Lipídico Tipo de estudo: Observational_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged / Newborn Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2019 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Contexto em Saúde: 2_ODS3 / 3_ND / 4_TD / 6_ODS3_enfermedades_notrasmisibles / 7_ODS3_muertes_prevenibles_nacidos_ninos Base de dados: MEDLINE Assunto principal: Oxirredução / Triglicerídeos / Ácidos Graxos / Erros Inatos do Metabolismo Lipídico Tipo de estudo: Observational_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged / Newborn Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2019 Tipo de documento: Article