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A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.
Truxal, K V; Fu, H; McCarty, D M; McNally, K A; Kunkler, K L; Zumberge, N A; Martin, L; Aylward, S C; Alfano, L N; Berry, K M; Lowes, L P; Corridore, M; McKee, C; McBride, K L; Flanigan, K M.
Afiliação
  • Truxal KV; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Division of Molecular and Human Genetics, Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbus, OH, United States.
  • Fu H; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbus, OH, United States.
  • McCarty DM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbus, OH, United States.
  • McNally KA; Center for Biobehavioral Outcomes Core, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Division of Neuropsychology, Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbus, OH, United States.
  • Kunkler KL; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
  • Zumberge NA; Division of Radiology, Nationwide Children's Hospital, Columbus, OH, United States; Department of Radiology, The Ohio State University, Columbus, OH, United States.
  • Martin L; Division of Radiology, Nationwide Children's Hospital, Columbus, OH, United States; Department of Radiology, The Ohio State University, Columbus, OH, United States.
  • Aylward SC; Division of Neurology, Nationwide Children's Hospital, Columbus, OH, United States; Department of Neurology, The Ohio State University, Columbus, OH, United States.
  • Alfano LN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
  • Berry KM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
  • Lowes LP; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States.
  • Corridore M; Division of Anesthesiology, Nationwide Children's Hospital, Columbus, OH, United States; Department ofAnesthesiology, The Ohio State University, Columbus, OH, United States.
  • McKee C; Division of Anesthesiology, Nationwide Children's Hospital, Columbus, OH, United States.
  • McBride KL; Center for Cardiovascular and Pulmonary Research, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Division of Molecular and Human Genetics, Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbu
  • Flanigan KM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States; Division of Neurology, Nationwide Children's Hospital, Columbus, OH, United States; Department of Pediatrics, The Ohio State University, Columbus, OH, United States; Department of Neurolog
Mol Genet Metab ; 119(3): 239-248, 2016 11.
Article em En | MEDLINE | ID: mdl-27590925
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Limite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2016 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Clinical_trials Limite: Child / Child, preschool / Female / Humans / Infant / Male Idioma: En Ano de publicação: 2016 Tipo de documento: Article