The failed clinical story of myostatin inhibitors against Duchenne muscular dystrophy: exploring the biology behind the battle

E Rybalka, CA Timpani, DA Debruin, RM Bagaric… - Cells, 2020 - mdpi.com
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 …

A mouse anti-myostatin antibody increases muscle mass and improves muscle strength and contractility in the mdx mouse model of Duchenne muscular dystrophy …

M St. Andre, M Johnson, PN Bansal, J Wellen… - Skeletal muscle, 2017 - Springer
Background The treatments currently approved for Duchenne muscular dystrophy (DMD), a
progressive skeletal muscle wasting disease, address the needs of only a small proportion …

Pharmacological strategies for muscular dystrophy

TS Khurana, KE Davies - Nature Reviews Drug Discovery, 2003 - nature.com
Duchenne muscular dystrophy (DMD) is a fatal, genetic disorder whose relentless
progression underscores the urgency for developing a cure. Although Duchenne initiated …

Myostatin propeptide‐mediated amelioration of dystrophic pathophysiology

S Bogdanovich, KJ Perkins, TOB Krag… - The FASEB …, 2005 - Wiley Online Library
Mutations in myostatin (GDF8) cause marked increases in muscle mass, suggesting that this
transforming growth factor‐β (TGF‐β) superfamily member negatively regulates muscle …

[HTML][HTML] The elusive promise of myostatin inhibition for muscular dystrophy

KR Wagner - Current opinion in neurology, 2020 - journals.lww.com
The elusive promise of myostatin inhibition for muscular dys... : Current Opinion in Neurology
The elusive promise of myostatin inhibition for muscular dystrophy : Current Opinion in …

[HTML][HTML] Glucocorticoids counteract hypertrophic effects of myostatin inhibition in dystrophic muscle

DW Hammers, CC Hart, A Patsalos, MK Matheny… - JCI insight, 2020 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is a devastating genetic muscle disease resulting in
progressive muscle degeneration and wasting. Glucocorticoids, specifically prednisone …

Antimyostatin treatment in health and disease: The story of great expectations and limited success

TL Nielsen, J Vissing, TO Krag - Cells, 2021 - mdpi.com
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 …

Interference with myostatin/ActRIIB signaling as a therapeutic strategy for Duchenne muscular dystrophy

H Amthor, W MH Hoogaars - Current gene therapy, 2012 - ingentaconnect.com
Since the discovery of the myostatin/ActRIIB signaling pathway 15 years ago, numerous
strategies were developed to block its inhibitory function during skeletal muscle growth …

Systemic myostatin inhibition via liver-targeted gene transfer in normal and dystrophic mice

KJ Morine, LT Bish, K Pendrak, MM Sleeper… - PloS one, 2010 - journals.plos.org
Background Myostatin inhibition is a promising therapeutic strategy to maintain muscle mass
in a variety of disorders, including the muscular dystrophies, cachexia, and sarcopenia …

Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

V Mariot, R Joubert, C Hourdé, L Féasson… - Nature …, 2017 - nature.com
Muscular dystrophies are characterized by weakness and wasting of skeletal muscle
tissues. Several drugs targeting the myostatin pathway have been used in clinical trials to …