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Significance Duchenne muscular dystrophy (DMD) is a fatal degenerative disease without a cure. Current standard pharmacological treatment is corticosteroids. Their prolonged use is associated with several undesirable side effects. Using Caenorhabditis elegans , we have identified pharmacological treatments that supplement hydrogen sulfide (H 2 S). One, sodium GYY4137, largely acts like prednisone to improve neuromuscular health; the other, AP39, targets H 2 S delivery to mitochondria. As these are not steroids, they are unlikely to produce steroid-induced side effects. Additionally, as DMD mice show a decline in total sulfide, our results pave the way for evaluation of cellular and/or mitochondrial H 2 S in DMD pathology and warrant further investigation of selective H 2 S delivery approaches in mdx mice and/or higher animal models of DMD.

Original publication

DOI

10.1073/pnas.2018342118

Type

Journal article

Journal

Proceedings of the National Academy of Sciences

Publisher

Proceedings of the National Academy of Sciences

Publication Date

02/03/2021

Volume

118