Why the study?
Following variable behavioral benefit from myostatin inhibition in Acta1 mutant models, this study evaluated the effects of ActRIIB-mFc in a nebulin conditional knockout model of nemaline myopathy.
Does ActRIIB-mFc improve weight gain, strength, myofiber size, or hypertrophic pathway signaling in the Neb cKO mouse model of nemaline myopathy?
Population
Nebulin conditional knockout (Neb cKO) mouse model of nemaline myopathy
Comparison
ActRIIB-mFc treatment
Authors
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No benefit from myostatin inhibition in this nebulin model; leaves open efficacy in other nemaline myopathy subtypes or patients.
Does ActRIIB-mFc improve weight gain, strength, myofiber size, or hypertrophic pathway signaling in the Neb cKO mouse model of nemaline myopathy?
Myostatin inhibition with ActRIIB-mFc does not provide therapeutic benefit in the nebulin conditional knockout mouse model of nemaline myopathy, contrasting with effects seen in other models.
Tinklenberg et al. (2018) studied this question.
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