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A phase 1/2 open label nonrandomized clinical trial of intravenous 2-hydroxypropyl-ß-cyclodextrin for acute liver disease in infants with Niemann-Pick C1.
Reynolds, Margaret; Linneman, Laura A; Luna, Sofia; Warner, Barbara B; Turmelle, Yumirle P; Kulkarni, Sakil S; Jiang, Xuntian; Khanna, Geetika; Shinawi, Marwan; Porter, Forbes D; Ory, Daniel S; Cole, F Sessions; Dickson, Patricia I.
Afiliação
  • Reynolds M; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Linneman LA; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Luna S; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Warner BB; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Turmelle YP; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Kulkarni SS; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Jiang X; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Khanna G; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Shinawi M; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Porter FD; Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH, DHHS, Bethesda, MD, United States of America.
  • Ory DS; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Cole FS; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
  • Dickson PI; Washington University in St. Louis School of Medicine, St. Louis, MO, United States of America.
Mol Genet Metab Rep ; 28: 100772, 2021 Sep.
Article em En | MEDLINE | ID: mdl-34113546
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Revista: Mol Genet Metab Rep Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Guideline Idioma: En Revista: Mol Genet Metab Rep Ano de publicação: 2021 Tipo de documento: Article