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Acute and chronic tirasemtiv treatment improves in vivo and in vitro muscle performance in actin-based nemaline myopathy mice.
de Winter, Josine M; Gineste, Charlotte; Minardi, Elisa; Brocca, Lorenza; Rossi, Maira; Borsboom, Tamara; Beggs, Alan H; Bernard, Monique; Bendahan, David; Hwee, Darren T; Malik, Fady I; Pellegrino, Maria Antonietta; Bottinelli, Roberto; Gondin, Julien; Ottenheijm, Coen A C.
Afiliação
  • de Winter JM; Department of Physiology, Amsterdam UMC (location VUmc), Amsterdam 1081 HV, The Netherlands.
  • Gineste C; Aix-Marseille Univ, CNRS, CRMBM, UMR 7339, 13005 Marseille, France.
  • Minardi E; Department of Molecular Medicine, University of Pavia, Pavia 27100, Italy.
  • Brocca L; Department of Molecular Medicine, University of Pavia, Pavia 27100, Italy.
  • Rossi M; Department of Molecular Medicine, University of Pavia, Pavia 27100, Italy.
  • Borsboom T; Department of Physiology, Amsterdam UMC (location VUmc), Amsterdam 1081 HV, The Netherlands.
  • Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Bernard M; Aix-Marseille Univ, CNRS, CRMBM, UMR 7339, 13005 Marseille, France.
  • Bendahan D; Aix-Marseille Univ, CNRS, CRMBM, UMR 7339, 13005 Marseille, France.
  • Hwee DT; Research and Early Development, Cytokinetics Inc., South San Francisco, CA 94080, USA.
  • Malik FI; Research and Early Development, Cytokinetics Inc., South San Francisco, CA 94080, USA.
  • Pellegrino MA; Department of Molecular Medicine, University of Pavia, Pavia 27100, Italy.
  • Bottinelli R; Interdipartimental Centre for Biology and Sport Medicine, University of Pavia, Pavia 27100, Italy.
  • Gondin J; Department of Molecular Medicine, University of Pavia, Pavia 27100, Italy.
  • Ottenheijm CAC; Istituti Clinici Maugeri (IRCCS), Scientific Institute of Pavia, Pavia 27100, Italy.
Hum Mol Genet ; 30(14): 1305-1320, 2021 06 26.
Article em En | MEDLINE | ID: mdl-33909041
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Actinas / Miopatias da Nemalina Tipo de estudo: Guideline / Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Actinas / Miopatias da Nemalina Tipo de estudo: Guideline / Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article