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Table 1 Comprehensive overview of studies using agents to blunt transforming growth factor (TGF)-β signaling

From: Role of TGF-β signaling in inherited and acquired myopathies

Compound Mechanism of action Clinical condition Model organism Phenotypic findings Ref
FDA-approved medications
Losartan AT1a receptor antagonist (mostly used for hypertension, cardiomyopathies) MFSb Fbn1C1039G/+ mice Improved muscle architecture, function and regeneration [54]
   DMDc mdx mice Improved skeletal, diaphragmatic and cardiac muscle architecture, function and regeneration [54, 55]
   Muscle Injury Younge mice Decreased fibrosis and improved regeneration [56]
Suramin TGF-β1 receptor antagonist (anti-parasitic, anti-neoplasic) DMD mdx mice Decreased fibrosis and prevented decrease in grip strength [57]
Anti-fibrotic agents
Decorin Binds to TGF-β1 ligands Muscle injury Young mice Decreased fibrosis, improved regeneration and functional recovery [61]
   DMD mdx mice Decreased collagen type I levels in diaphragm [62]
γ-Interferon Induces Smad7 expression Muscle injury Young mice Decreased fibrosis, improved regeneration and functional recovery [63]
Pirfenidone TGF-β1 antagonist DMD mdx mice Improved cardiac function, minor alterations on the development of fibrosis, and no improvement in diaphragmatic function [64, 65]
Halofuginone Inhibits TGF-β-dependent phosphorylation of Smad3 DMD mdx mice Decreased fibrosis and improved function of the heart,diaphragm and limb muscles [66, 67]
   CMDd dy 2J /dy 2J Decreased fibrosis and improved functional performance but did not improve strength [68]
TGF-β neutralizing antibody
Neutralizes TGF-β (1 and/or 2) ligands MFS Fbn1C1039G/+ mice Prevented muscle atrophy and improved regeneration [54]
   DMD mdx mice Decreased fibrosis and improved regeneration [54, 69]
   Sarcopenia Agedg mice Failed to improve regeneration [70]
TGF-β receptor kinase inhibitor
Decoy receptor composed of extracellular portion of TGF-β receptor II Sarcopenia Aged mice Improved regeneration after direct intramuscular injection [70]
  1. aAngiotensin II type 1 receptor.
  2. bMarfan syndrome.
  3. cDuchenne muscular dystrophy.
  4. dCongenital muscular dystrophy.
  5. eAge ≤ 3 months.
  6. fAge 3-15 months.
  7. gAge ≥ 15 months.