DMD – ANIMAL MODELS: EP.88 Inflammasome inhibitors for the treatment of muscular dystrophies

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Duchenne muscular dystrophy (DMD) is the most frequent inherited human myopathy and one of the most devastating muscular dystrophies. Although dystrophin mutations represent the primary cause of DMD, it is the secondary processes involving persistent inflammation that likely exacerbate disease progression. Our group previously described the involvement of the NLRP3 inflammasome as having a major role in the deleterious inflammatory process worsening the dystrophic phenotype. Recently, MCC950 was discovered as an extremely potent, selective, small molecule inhibitors of NLRP3 and could thus be promising in muscle diseases with an inflammatory component.