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The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle

1
Institute for Health and Sport (IHeS), Victoria University, Melbourne, Victoria 8001, Australia
2
Australian Institute for Musculoskeletal Science (AIMSS), Victoria University, St Albans, Victoria 3021, Australia
3
Department of Medicine—Western Health, Melbourne Medical School, The University of Melbourne, Melbourne, Victoria 3021, Australia
*
Authors to whom correspondence should be addressed.
Cells 2020, 9(12), 2657; https://doi.org/10.3390/cells9122657
Received: 23 November 2020 / Revised: 8 December 2020 / Accepted: 9 December 2020 / Published: 10 December 2020
(This article belongs to the Special Issue Myostatin in Aging and Disease)
Myostatin inhibition therapy has held much promise for the treatment of muscle wasting disorders. This is particularly true for the fatal myopathy, Duchenne Muscular Dystrophy (DMD). Following on from promising pre-clinical data in dystrophin-deficient mice and dogs, several clinical trials were initiated in DMD patients using different modality myostatin inhibition therapies. All failed to show modification of disease course as dictated by the primary and secondary outcome measures selected: the myostatin inhibition story, thus far, is a failed clinical story. These trials have recently been extensively reviewed and reasons why pre-clinical data collected in animal models have failed to translate into clinical benefit to patients have been purported. However, the biological mechanisms underlying translational failure need to be examined to ensure future myostatin inhibitor development endeavors do not meet with the same fate. Here, we explore the biology which could explain the failed translation of myostatin inhibitors in the treatment of DMD. View Full-Text
Keywords: myostatin inhibition; Duchenne Muscular Dystrophy; skeletal muscle; muscle development; clinical trials; translation myostatin inhibition; Duchenne Muscular Dystrophy; skeletal muscle; muscle development; clinical trials; translation
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MDPI and ACS Style

Rybalka, E.; Timpani, C.A.; Debruin, D.A.; Bagaric, R.M.; Campelj, D.G.; Hayes, A. The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle. Cells 2020, 9, 2657. https://doi.org/10.3390/cells9122657

AMA Style

Rybalka E, Timpani CA, Debruin DA, Bagaric RM, Campelj DG, Hayes A. The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle. Cells. 2020; 9(12):2657. https://doi.org/10.3390/cells9122657

Chicago/Turabian Style

Rybalka, Emma; Timpani, Cara A.; Debruin, Danielle A.; Bagaric, Ryan M.; Campelj, Dean G.; Hayes, Alan. 2020. "The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle" Cells 9, no. 12: 2657. https://doi.org/10.3390/cells9122657

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Note that from the first issue of 2016, MDPI journals use article numbers instead of page numbers. See further details here.

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