Acute and chronic tirasemtiv treatment improves in vivo and in vitro muscle performance in actin-based nemaline myopathy mice

Nemaline myopathy, a disease of the actin-based thin filament, is one of the most frequent congenital myopathies. To date, no specific therapy is available to treat muscle weakness in nemaline myopathy. We tested the ability of tirasemtiv, a fast skeletal troponin activator that targets the thin fil...

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Publicado en:Hum Mol Genet
Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: Oxford University Press 2021
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC8255131/
https://ncbi.nlm.nih.gov/pubmed/33909041
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddab112
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