Article
Acute and chronic tirasemtiv treatment improves in vivo and in vitro muscle performance in actin-based nemaline myopathy mice.
Human molecular genetics - 26 Jun 2021
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
Abstract excerpt
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 filament, to augment muscle force-both in vivo and in vitro-in a nemaline myopathy mouse model with a mutation (H40Y)...
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