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Comment
. 2023 Mar 7;120(10):e2300988120.
doi: 10.1073/pnas.2300988120. Epub 2023 Feb 27.

Rare disease informs mechanism and possible treatment of statin-associated myopathy

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Comment

Rare disease informs mechanism and possible treatment of statin-associated myopathy

Tamar Harel. Proc Natl Acad Sci U S A. .
No abstract available

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Conflict of interest statement

The author declares no competing interest.

Figures

Fig. 1.
Fig. 1.
HMG-CoA reductase dysfunction by genetic, environmental, or autoimmune mechanisms. A biallelic variant in HMGCR, encoding the rate-limiting enzyme in cholesterol synthesis known as HMG-CoA reductase, is herein reported to cause limb-girdle muscular dystrophy (LGMD). The disease responds to treatment with a derivative of mevalonate, which is the downstream metabolite of HMG-CoA reductase. The Middle panel indicates the effect of statins as inhibitors of HMG-CoA reductase, with resultant myopathy as a common adverse effect. The Right panel demonstrates anti-HMGCR myositis, which can result from auto-antibodies to HMG-CoA reductase triggered by or independent of statin exposure.

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