Characterising and evaluating the efficacy of metabolic therapies for the treatment of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a fatal skeletal muscle wasting disease underpinned by extensive metabolic dysfunction. This culminates in reduced energy production which is detrimental to dystrophic muscle since buffering damage and stimulating repair are energy dependent processes. Current treatment options for DMD are limited and do not address this metabolic dysfunction despite the extensive role it plays in DMD pathogenesis and disease progression. Therefore, this thesis investigated the efficacy of two metabolic therapies, sodium nitrate and adenylosuccinic acid (ASA), to improve skeletal muscle metabolism and architecture in the well-established mdx mouse model of DMD, and in immortalised myoblasts derived from DMD patients