Therapeutic developments for Duchenne muscular dystrophy

IEC Verhaart, A Aartsma-Rus - Nature Reviews Neurology, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Exercise is the real polypill

C Fiuza-Luces, N Garatachea, NA Berger… - Physiology, 2013 - journals.physiology.org
The concept of a “polypill” is receiving growing attention to prevent cardiovascular disease.
Yet similar if not overall higher benefits are achievable with regular exercise, a drug-free …

Drug development progress in duchenne muscular dystrophy

J Deng, J Zhang, K Shi, Z Liu - Frontiers in Pharmacology, 2022 - frontiersin.org
Duchenne muscular dystrophy (DMD) is a severe, progressive, and incurable X-linked
disorder caused by mutations in the dystrophin gene. Patients with DMD have an absence of …

Myostatin/activin pathway antagonism: molecular basis and therapeutic potential

HQ Han, X Zhou, WE Mitch, AL Goldberg - The international journal of …, 2013 - Elsevier
Muscle wasting is associated with a wide range of catabolic diseases. This debilitating loss
of muscle mass and functional capacity reduces the quality of life and increases the risks of …

Role of TGF-β signaling in inherited and acquired myopathies

TN Burks, RD Cohn - Skeletal muscle, 2011 - Springer
The transforming growth factor-beta (TGF-β) superfamily consists of a variety of cytokines
expressed in many different cell types including skeletal muscle. Members of this …

[HTML][HTML] Myostatin inhibitors as therapies for muscle wasting associated with cancer and other disorders

RC Smith, BK Lin - Current opinion in supportive and palliative …, 2013 - journals.lww.com
Myostatin inhibitors as therapies for muscle wasting associa... : Current Opinion in Supportive
and Palliative Care Myostatin inhibitors as therapies for muscle wasting associated with cancer …

IRE1α regulates skeletal muscle regeneration through myostatin mRNA decay

S He, T Fu, Y Yu, Q Liang, L Li, J Liu… - The Journal of …, 2021 - Am Soc Clin Investig
Skeletal muscle can undergo a regenerative process in response to injury or disease to
preserve muscle mass and function, which are critically influenced by cellular stress …

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 …

Thrown for a loop: fibro-adipogenic progenitors in skeletal muscle fibrosis

T Loomis, LR Smith - American Journal of Physiology-Cell …, 2023 - journals.physiology.org
Fibro-adipogenic progenitors (FAPs) are key regulators of skeletal muscle regeneration and
homeostasis. However, dysregulation of these cells leads to fibro-fatty infiltration across …

Myostatin from the heart: local and systemic actions in cardiac failure and muscle wasting

A Breitbart, M Auger-Messier… - American Journal of …, 2011 - journals.physiology.org
A significant proportion of heart failure patients develop skeletal muscle wasting and cardiac
cachexia, which is associated with a very poor prognosis. Recently, myostatin, a cytokine …