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2-Hydroxypropyl-ß-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.
Davidson, Jessica; Molitor, Elizabeth; Moores, Samantha; Gale, Sarah E; Subramanian, Kanagaraj; Jiang, Xuntian; Sidhu, Rohini; Kell, Pamela; Zhang, Jesse; Fujiwara, Hideji; Davidson, Cristin; Helquist, Paul; Melancon, Bruce J; Grigalunas, Michael; Liu, Gang; Salahi, Farbod; Wiest, Olaf; Xu, Xin; Porter, Forbes D; Pipalia, Nina H; Cruz, Dana L; Holson, Edward B; Schaffer, Jean E; Walkley, Steven U; Maxfield, Frederick R; Ory, Daniel S.
Afiliación
  • Davidson J; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Molitor E; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Moores S; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Gale SE; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Subramanian K; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Jiang X; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Sidhu R; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Kell P; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Zhang J; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Fujiwara H; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Davidson C; Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center, Albert Einstein College of Medicine, 1410 Pelham Parkway South, Bronx, NY 10461, USA.
  • Helquist P; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Melancon BJ; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Grigalunas M; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Liu G; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Salahi F; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Wiest O; Department of Chemistry and Biochemistry, University of Notre Dame, Notre Dame, IN 46556 5670, USA.
  • Xu X; Division of Preclinical Innovation, National Center for Advancing Translational Sciences (NCATS), 9800 Medical Center Dr., National Institutes of Health, Rockville, MD 20850, USA.
  • Porter FD; Section on Molecular Dysmorphology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, NIH, DHHS, Bethesda, MD 20892, USA.
  • Pipalia NH; Department of Biochemistry, Weill Cornell Medical College, New York, NY 10065, USA.
  • Cruz DL; Department of Biochemistry, Weill Cornell Medical College, New York, NY 10065, USA.
  • Holson EB; KDac Therapeutics, Cambridge, MA 02139, USA.
  • Schaffer JE; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA.
  • Walkley SU; Dominick P. Purpura Department of Neuroscience, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center, Albert Einstein College of Medicine, 1410 Pelham Parkway South, Bronx, NY 10461, USA.
  • Maxfield FR; Department of Biochemistry, Weill Cornell Medical College, New York, NY 10065, USA.
  • Ory DS; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA. Electronic address: dory@wustl.edu.
Biochim Biophys Acta Mol Cell Biol Lipids ; 1864(10): 1545-1561, 2019 10.
Article en En | MEDLINE | ID: mdl-31051283
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Polietilenglicoles / Enfermedad de Niemann-Pick Tipo C / Inhibidores de Histona Desacetilasas / 2-Hidroxipropil-beta-Ciclodextrina / Vorinostat Límite: Animals Idioma: En Revista: Biochim Biophys Acta Mol Cell Biol Lipids Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
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XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Polietilenglicoles / Enfermedad de Niemann-Pick Tipo C / Inhibidores de Histona Desacetilasas / 2-Hidroxipropil-beta-Ciclodextrina / Vorinostat Límite: Animals Idioma: En Revista: Biochim Biophys Acta Mol Cell Biol Lipids Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos