Key result
Inhibition of myostatin via follistatin overexpression or ActRIIB-Fc administration did not improve muscle mass, motor function, or survival in severe spinal muscular atrophy mice.
Why the study?
Does inhibition of myostatin signaling improve muscle mass, motor function, or survival in severe spinal muscular atrophy mice?
Population
Severe spinal muscular atrophy (SMA) mice (hSMN2/delta7SMN/mSmn(-/-))
Comparison
Inhibition of myostatin signaling via transgenic… vs Vehicle-treated mice (for the ActRIIB-Fc group)
Design
Preclinical
Authors
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Myostatin inhibition lacks benefit in severe SMA mice; leaves open utility in milder forms or other myopathies.
Does inhibition of myostatin signaling improve muscle mass, motor function, or survival in severe spinal muscular atrophy mice?
Inhibition of myostatin does not appear to be a promising therapeutic strategy in severe forms of spinal muscular atrophy based on mouse models.
Sumner et al. (2009) studied severe spinal muscular atrophy. myostatin inhibition (follistatin overexpression or ActRIIB-Fc) vs. vehicle was evaluated on muscle mass, motor function, and survival. Inhibition of myostatin via follistatin overexpression or ActRIIB-Fc administration did not improve muscle mass, motor function, or survival in severe spinal muscular atrophy mice.
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