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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Vydáno v:Hum Mol Genet
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2021
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General Article
On-line přístup: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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