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.