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Mifepristone boosts survival, muscle size in SMA mouse model
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Mifepristone boosts survival, muscle size in SMA mouse model
Mifepristone, a glucocorticoid blocker, increased survival and muscle size in a mouse model of spinal muscular atrophy (SMA), according to a recent study. Combining mifepristone with a gene therapy construct similar to the one used in Zolgensma and Itvisma, which is intended to increase levels of the SMN protein, improved tissue- and sex-specific responses to treatment. Most SMA cases result in an inadequate amount of SMN, a key protein for the …
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