Myotubular myopathy is a rare disease of genetic origin characterized by significant muscle weakness leading to respiratory disorders and for which no treatment exists today. In this paper, we show that inhibition of the activity of the enzyme PI3KC2β prevents the development of this myopathy in a mouse model of the disease, thus identifying a therapeutic target to treat myotubular myopathy in humans.
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| http://dx.doi.org/10.1051/medsci/2023134 | DOI Listing |
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