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The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
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Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success

Copenhagen Neuromuscular Center, Department of Neurology, Rigshospitalet, University of Copenhagen, DK-2100 Copenhagen, Denmark
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Academic Editor: Christopher Fry
Cells 2021, 10(3), 533; https://doi.org/10.3390/cells10030533
Received: 9 February 2021 / Revised: 24 February 2021 / Accepted: 26 February 2021 / Published: 3 March 2021
(This article belongs to the Special Issue Myostatin in Aging and Disease)
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. View Full-Text
Keywords: myostatin; muscular dystrophy; muscular regeneration; ActRIIB; TGF-β myostatin; muscular dystrophy; muscular regeneration; ActRIIB; TGF-β
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MDPI and ACS Style

Nielsen, T.L.; Vissing, J.; Krag, T.O. Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success. Cells 2021, 10, 533. https://doi.org/10.3390/cells10030533

AMA Style

Nielsen TL, Vissing J, Krag TO. Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success. Cells. 2021; 10(3):533. https://doi.org/10.3390/cells10030533

Chicago/Turabian Style

Nielsen, Tue L.; Vissing, John; Krag, Thomas O. 2021. "Antimyostatin Treatment in Health and Disease: The Story of Great Expectations and Limited Success" Cells 10, no. 3: 533. https://doi.org/10.3390/cells10030533

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