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

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 ini...
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Autor*in:	Emma Rybalka [verfasserIn] Cara A. Timpani [verfasserIn] Danielle A. Debruin [verfasserIn] Ryan M. Bagaric [verfasserIn] Dean G. Campelj [verfasserIn] Alan Hayes [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2020

Schlagwörter:	myostatin inhibition Duchenne Muscular Dystrophy skeletal muscle muscle development clinical trials translation

Übergeordnetes Werk:	In: Cells - MDPI AG, 2012, 9(2020), 12, p 2657
Übergeordnetes Werk:	volume:9 ; year:2020 ; number:12, p 2657

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.3390/cells9122657

Katalog-ID:	DOAJ052853470

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language	English
source	In Cells 9(2020), 12, p 2657 volume:9 year:2020 number:12, p 2657
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institution	findex.gbv.de
topic_facet	myostatin inhibition Duchenne Muscular Dystrophy skeletal muscle muscle development clinical trials translation Cytology
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authorswithroles_txt_mv	Emma Rybalka @@aut@@ Cara A. Timpani @@aut@@ Danielle A. Debruin @@aut@@ Ryan M. Bagaric @@aut@@ Dean G. Campelj @@aut@@ Alan Hayes @@aut@@
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author	Emma Rybalka
spellingShingle	Emma Rybalka misc QH573-671 misc myostatin inhibition misc Duchenne Muscular Dystrophy misc skeletal muscle misc muscle development misc clinical trials misc translation misc Cytology The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
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topic_title	QH573-671 The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle myostatin inhibition Duchenne Muscular Dystrophy skeletal muscle muscle development clinical trials translation
topic	misc QH573-671 misc myostatin inhibition misc Duchenne Muscular Dystrophy misc skeletal muscle misc muscle development misc clinical trials misc translation misc Cytology
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title	The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
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title_auth	The Failed Clinical Story of Myostatin Inhibitors against Duchenne Muscular Dystrophy: Exploring the Biology behind the Battle
abstract	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.
abstractGer	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.
abstract_unstemmed	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.
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