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Clinical potential of microdystrophin as a surrogate endpoint.
Boehler, Jessica F; Brown, Kristy J; Beatka, Margaret; Gonzalez, J Patrick; Donisa Dreghici, Roxana; Soustek-Kramer, Meghan; McGonigle, Sharon; Ganot, Annie; Palmer, Timothy; Lowie, Caitlin; Chamberlain, Jeffrey S; Lawlor, Michael W; Morris, Carl A.
Afiliação
  • Boehler JF; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Brown KJ; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Beatka M; Diverge TSL, 247 Freshwater Way Suite 610, Milwaukee, WI 53204, United States.
  • Gonzalez JP; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Donisa Dreghici R; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Soustek-Kramer M; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • McGonigle S; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Ganot A; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Palmer T; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Lowie C; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States.
  • Chamberlain JS; Department of Neurology, University of Washington School of Medicine, Seattle, United States.
  • Lawlor MW; Diverge TSL, 247 Freshwater Way Suite 610, Milwaukee, WI 53204, United States.
  • Morris CA; Solid Biosciences, 500 Rutherford Avenue 3rd Floor, Boston, MA 02129, United States. Electronic address: carl@solidbio.com.
Neuromuscul Disord ; 33(1): 40-49, 2023 01.
Article em En | MEDLINE | ID: mdl-36575103
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article