DMD – ANIMAL MODELS & PRECLINICAL TREATMENT

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In DMD, NF-kB is persistently upregulated in cardiac and skeletal muscle from infancy, driving inflammation and fibrosis, and potentially leading to myocardial fibrosis and dysfunction. Therapies to drive reverse cardiac remodelling and improve cardiac function in DMD are limited. Edasalonexent is an oral small molecule NF-kB inhibitor in Phase 3 clinical development. We hypothesized that edasalonexent could prevent cardiac dysfunction in mdx: Utrn+/- mice, a model of DMD in which cardiac dysfunction develops by 6-8 weeks of age.

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