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Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Babcock, Michael C; Mikulka, Christina R; Wang, Bing; Chandriani, Sanjay; Chandra, Sundeep; Xu, Yue; Webster, Katherine; Feng, Ying; Nelvagal, Hemanth R; Giaramita, Alex; Yip, Bryan K; Lo, Melanie; Jiang, Xuntian; Chao, Qi; Woloszynek, Josh C; Shen, Yuqiao; Bhagwat, Shripad; Sands, Mark S; Crawford, Brett E.
  • Babcock MC; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Mikulka CR; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Wang B; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Chandriani S; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Chandra S; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Xu Y; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Webster K; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Feng Y; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Nelvagal HR; Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Giaramita A; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Yip BK; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Lo M; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Jiang X; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Chao Q; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Woloszynek JC; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Shen Y; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Bhagwat S; BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA, 94949, USA.
  • Sands MS; Department of Medicine, Washington University School of Medicine, St. Louis, MO, 63110, USA.
  • Crawford BE; Department of Genetics, Washington University School of Medicine, St. Louis, MO, 63110, USA.
Sci Rep ; 11(1): 14486, 2021 07 14.
Article en En | MEDLINE | ID: mdl-34262084
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Inhibidores Enzimáticos / N-Acilesfingosina Galactosiltransferasa / Leucodistrofia de Células Globoides / Leucodistrofia Metacromática Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Inhibidores Enzimáticos / N-Acilesfingosina Galactosiltransferasa / Leucodistrofia de Células Globoides / Leucodistrofia Metacromática Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article