Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success

In the past 20 years, myostatin, a negative regulator of muscle mass, has attracted attention as a potential therapeutic target in muscular dystrophies and other conditions. Preclinical studies have shown potential for increasing muscular mass and ameliorating the pathological features of dystrophic muscle by the inhibition of myostatin in various ways. However, hardly any clinical trials have proven to translate the promising results from the animal models into patient populations. We present the background for myostatin regulation, clinical and preclinical results and discuss why translation from animal models to patients is difficult. Based on this, we put the clinical relevance of future antimyostatin treatment into perspective.

Automated summary

While preclinical studies have shown potential for increasing muscle mass and improving the pathological features of muscle diseases by inhibiting myostatin, there has been a lack of successful translation of these results into clinical trials with patient populations.