Journal article

Nitric Oxide (NO) and Duchenne Muscular Dystrophy: NO Way to Go?

Cara A Timpani, Kamel Mamchaoui, Gillian Butler-Browne, Emma Rybalka

ANTIOXIDANTS | MDPI | Published : 2020


The discordance between pre-clinical success and clinical failure of treatment options for Duchenne Muscular Dystrophy (DMD) is significant. The termination of clinical trials investigating the phosphodiesterase inhibitors, sildenafil and tadalafil (which prolong the second messenger molecule of nitric oxide (NO) signaling), are prime examples of this. Both attenuated key dystrophic features in the mdx mouse model of DMD yet failed to modulate primary outcomes in clinical settings. We have previously attempted to modulate NO signaling via chronic nitrate supplementation of the mdx mouse but failed to demonstrate beneficial modulation of key dystrophic features (i.e., metabolism). Instead, we..

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University of Melbourne Researchers