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Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1.
Maarup, Timothy J; Chen, Agnes H; Porter, Forbes D; Farhat, Nicole Y; Ory, Daniel S; Sidhu, Rohini; Jiang, Xuntian; Dickson, Patricia I.
Afiliación
  • Maarup TJ; Department of Pediatrics, Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA. Electronic address: tmaarup@labiomed.org.
  • Chen AH; Department of Pediatrics, Division of Neurology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
  • Porter FD; Program in Developmental Endocrinology and Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, USA.
  • Farhat NY; Program in Developmental Endocrinology and Genetics, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, DHHS, Bethesda, MD, USA.
  • Ory DS; Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St Louis, MO, USA.
  • Sidhu R; Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St Louis, MO, USA.
  • Jiang X; Diabetic Cardiovascular Disease Center, Washington University School of Medicine, St Louis, MO, USA.
  • Dickson PI; Department of Pediatrics, Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, USA.
Mol Genet Metab ; 116(1-2): 75-9, 2015.
Article en En | MEDLINE | ID: mdl-26189084
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inyecciones Espinales / Beta-Ciclodextrinas / Excipientes / Enfermedad de Niemann-Pick Tipo C Límite: Child / Humans / Male Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2015 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inyecciones Espinales / Beta-Ciclodextrinas / Excipientes / Enfermedad de Niemann-Pick Tipo C Límite: Child / Humans / Male Idioma: En Revista: Mol Genet Metab Asunto de la revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Año: 2015 Tipo del documento: Article Pais de publicación: Estados Unidos