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Neuromuscul Disord. 2015 Feb;25(2):111-9. doi: 10.1016/j.nmd.2014.10.002. Epub 2014 Oct 13.

Investigating sodium valproate as a treatment for McArdle disease in sheep.

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School of Veterinary and Life Sciences, Murdoch University, Perth, Western Australia, Australia; Australian Neuro-Muscular Research Institute, CNND, University of Western Australia, Perth, Western Australia, Australia. Electronic address:
School of Veterinary and Life Sciences, Murdoch University, Perth, Western Australia, Australia.
Centre for Neuromuscular Diseases, National Hospital for Neurology and Neurosurgery, London, United Kingdom.
Robert Jones and Agnes Hunt Orthopaedic hospital, Oswestry, United Kingdom.


McArdle disease is due to an absence of the enzyme muscle glycogen phosphorylase and results in significant physical impairment in humans. We hypothesised that sodium valproate, an HDAC inhibitor, might have the ability to up-regulate the enzyme. We treated McArdle sheep with sodium valproate given enterically at 20-60 mg/kg body wt. Compared with untreated control animals, there was increased expression of phosphorylase in muscle fibres. The response was dose dependent and reached a maximum 2 hours after the application and increased with repeated applications. Improvement in mobility could not be demonstrated. These findings suggest that sodium valproate is a potential therapeutic treatment for McArdle disease.


Glycogen phosphorylase; Glycogen storage disease type V; HDCAI; McArdle disease; Sheep; Sodium valproate

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