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Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial.
Í Dali, Christine; Sevin, Caroline; Krägeloh-Mann, Ingeborg; Giugliani, Roberto; Sakai, Norio; Wu, James; Wasilewski, Margaret.
Afiliação
  • Í Dali C; Department of Clinical Genetics, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark. Electronic address: cid@dadlnet.dk.
  • Sevin C; Neuropediatrics Unit, Bicêtre Hospital, Paris, France. Electronic address: caroline.sevin@inserm.fr.
  • Krägeloh-Mann I; Department of Neuropediatrics, University Children's Hospital Tübingen, Tübingen, Germany. Electronic address: ingeborg.kraegeloh-mann@med.uni-tuebingen.de.
  • Giugliani R; Medical Genetics Service, HCPA, Department of Genetics, UFRGS, and INAGEMP, Porto Alegre, Brazil. Electronic address: rgiugliani@hcpa.edu.br.
  • Sakai N; Osaka University Hospital, Osaka, Japan. Electronic address: norio@ped.med.osaka-u.ac.jp.
  • Wu J; Shire, a member of the Takeda group of companies, Lexington, MA, USA. Electronic address: james.wu100@yahoo.com.
  • Wasilewski M; Shire, a member of the Takeda group of companies, Lexington, MA, USA. Electronic address: mwasilewski@mac.com.
Mol Genet Metab ; 131(1-2): 235-244, 2020.
Article em En | MEDLINE | ID: mdl-32792226
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas Recombinantes / Terapia Genética / Cerebrosídeo Sulfatase / Leucodistrofia Metacromática Tipo de estudo: Prognostic_studies Limite: Adolescent / Animals / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Proteínas Recombinantes / Terapia Genética / Cerebrosídeo Sulfatase / Leucodistrofia Metacromática Tipo de estudo: Prognostic_studies Limite: Adolescent / Animals / Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article