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In addition, simvastatin has been taken by millions of people, so potential problems are clearly known. It’s possible that simvastatin could be a treatment on its own, slowing the progression of muscle degeneration.Most importantly, simvastatin is already approved for treatment of familial hyperlipidemia in the pediatric population, where clinical studies have shown a good safety profile. Also, a combination of simvastatin and other drugs currently being tested could be even more effective."Figure 2 Structure of mini- and microdystrophins and utrophin.
Our targeted pathogenic pathways, inflammation, oxidative stress (NOX2 expression) and fibrosis were all significantly reduced by simvastatin.
This translated into considerable improvement in muscle function, with a 40% increase in hind limb muscle force and resistance to muscle fatigue.
Minidystrophin is a dystrophin of mild BMD patients that is ~6.4 kb in size.
Based on this, the rod region was further truncated to create microdystrophin (4.9 kb) that can be incorporated into viral vectors.
Therefore, we postulated that if simvastatin reduced NOX2, as well as targeting inflammation and fibrosis, it would lead to improved muscle health and function in mice. Whitehead: In DMD muscles, the loss of dystrophin impacts many important signaling and homeostatic cellular functions in muscle fibres.
Our research has shown that oxidative stress and increased intracellular calcium entry through membrane ion channels stimulates degradative pathways that lead to muscle damage and cell death.
In this paper, I present the significance of molecular diagnosis and the development of mutation-based therapeutic strategies to complement or restore dystrophin expression.
View Full-Text Figure 1 Hypothetical pathogenic mechanism of dystrophin deficiency.
As a result, a truncated but functional dystrophin is produced, resulting in a mild dystrophic phenotype.
Whitehead: In addition to their well established cholesterol lowering benefits, statins also have potent anti-inflammatory, anti-fibrotic and antioxidant effects, which continue to be identified in a wide range of diseases.