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Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.
Kan, Shih-Hsin; Elsharkawi, Ibrahim; Le, Steven Q; Prill, Heather; Mangini, Linley; Cooper, Jonathan D; Lawrence, Roger; Sands, Mark S; Crawford, Brett E; Dickson, Patricia I.
Affiliation
  • Kan SH; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; CHOC Research Institute, Orange, CA 92868, United States of America. Electronic address: skan@choc.org.
  • Elsharkawi I; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of America.
  • Le SQ; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A
  • Prill H; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
  • Mangini L; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
  • Cooper JD; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A
  • Lawrence R; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
  • Sands MS; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of America.
  • Crawford BE; Biology Research, BioMarin Pharmaceutical Inc., Novato, CA 94949, United States of America.
  • Dickson PI; Department of Pediatrics, The Lundquist Institute (formally Los Angeles Biomedical Research Institute) at Harbor-UCLA Medical Center, Torrance, CA 90502, United States of America; Department of Pediatrics, Washington University School of Medicine in St. Louis, St. Louis, MO 63110, United States of A
Mol Genet Metab ; 133(2): 185-192, 2021 06.
Article de En | MEDLINE | ID: mdl-33839004
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Acetylglucosaminidase / Facteur de croissance IGF-II / Mucopolysaccharidose de type III / Thérapie enzymatique substitutive Type d'étude: Prognostic_studies Limites: Animals / Humans Langue: En Journal: Mol Genet Metab Sujet du journal: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Année: 2021 Type de document: Article
Texte intégral
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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Acetylglucosaminidase / Facteur de croissance IGF-II / Mucopolysaccharidose de type III / Thérapie enzymatique substitutive Type d'étude: Prognostic_studies Limites: Animals / Humans Langue: En Journal: Mol Genet Metab Sujet du journal: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Année: 2021 Type de document: Article