Edasalonexent (CAT-1004), an NF-kB inhibitor, enhances myotube formation in vitro, and increases exon-skipped sarcolemmal dystrophin in muscle of mdx mice

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NF-kB is activated from infancy in boys with Duchenne muscular dystrophy and mdx mice, and promotes muscle degeneration while inhibiting muscle regeneration. NF-kB driven micro-RNA also directly impair dystrophin protein translation, further destabilizing this protein and limiting the full potential of exon-skipping therapy in dystrophic muscles. Edasalonexent is an oral NF-kB inhibitor that is currently in the MoveDMD trial in DMD boys aged 4–7, an age range where high burden of inflammation in the muscle is expected.

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