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Edasalonexent is an orally administered small molecule drug designed to inhibit the transcription factor NF-kappa B (NF-κB), which plays a central role in inflammation, muscle degeneration, and suppression of muscle regeneration in Duchenne muscular dystrophy (DMD). Structurally, it is a bifunctional conjugate that covalently links salicylic acid and docosahexaenoic acid (DHA), both known NF-κB inhibitors. After cellular uptake, edasalonexent is hydrolyzed by fatty acid amide hydrolase (FAAH) to release its active components intracellularly. This dual delivery provides synergistic inhibition of NF-κB activity, reducing inflammation and fibrosis while improving muscle function. Edasalonexent was developed primarily for DMD but also investigated for dysferlinopathy and type 2 diabetes mellitus[2][3][4][5][6].
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