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Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease.
Koeberl, Dwight D; Case, Laura E; Smith, Edward C; Walters, Crista; Han, Sang-Oh; Li, Yanzhen; Chen, Wei; Hornik, Christoph P; Huffman, Kim M; Kraus, William E; Thurberg, Beth L; Corcoran, David L; Bali, Deeksha; Bursac, Nenad; Kishnani, Priya S.
Afiliação
  • Koeberl DD; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA. Electronic address: dwight.koeberl@duke.edu.
  • Case LE; Department of Physical and Occupational Therapy, Duke University School of Medicine, Durham, NC 27710, USA.
  • Smith EC; Division of Neurology, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  • Walters C; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  • Han SO; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  • Li Y; Department of Biomedical Engineering, Duke University School of Medicine, Durham, NC 27710, USA.
  • Chen W; Duke Center for Genomic and Computational Biology, Duke University School of Medicine, Durham, NC 27710, USA.
  • Hornik CP; Division of Critical Care Medicine, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  • Huffman KM; Division of Rheumatology and Immunology, Department of Medicine, Duke University School of Medicine, Durham, NC 27710, USA.
  • Kraus WE; Division of Cardiology, Department of Medicine; Duke University School of Medicine, Durham, NC 27710, USA.
  • Thurberg BL; Sanofi Genzyme, Cambridge, MA, USA.
  • Corcoran DL; Duke Center for Genomic and Computational Biology, Duke University School of Medicine, Durham, NC 27710, USA.
  • Bali D; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
  • Bursac N; Department of Biomedical Engineering, Duke University School of Medicine, Durham, NC 27710, USA.
  • Kishnani PS; Division of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, USA.
Mol Ther ; 26(9): 2304-2314, 2018 09 05.
Article em En | MEDLINE | ID: mdl-30025991
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Clembuterol / Músculo Esquelético Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2018 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II / Clembuterol / Músculo Esquelético Tipo de estudo: Clinical_trials / Prognostic_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2018 Tipo de documento: Article