Abstract
The main objective is to evaluate the efficacy of losmapimod in inhibiting the aberrant expression of DUX4, the root cause of FSHD. Secondary objectives are to evaluate the safety, tolerability, PK, and TE in blood and muscle, and muscle health with MRI. FSHD is caused by aberrant expression of DUX4 due to loss of repression at the D4Z4 locus. DUX4 activates a downstream transcriptional program that causes myofiber death, maladaptive tissue remodeling characterized by replacement of muscle with fat ultimately resulting in progressive motor disability.
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