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TL;DR
This narrative review discusses the pathophysiology and emerging pharmacotherapeutic strategies for muscular dystrophies, focusing on targeting chronic inflammation and fibrosis as alternatives to long-term glucocorticoid use.
Biomolecules · 6 authors, 2 centres
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This narrative review discusses the pathophysiology and emerging pharmacotherapeutic strategies for muscular dystrophies, focusing on targeting chronic inflammation and fibrosis as alternatives to long-term glucocorticoid use.
This narrative review summarizes the pathophysiology, genetic background, and emerging therapeutic strategies for muscular dystrophies. The authors highlight chronic inflammation and fibrosis as common pathological features and discuss the limitations of glucocorticoids, the current standard of care for Duchenne muscular dystrophy. They review preclinical and early clinical approaches targeting inflammatory pathways like NF-κB and TNF-α, including antioxidants, P2X7 antagonists, vamorolone, and rapamycin, as well as antifibrotic strategies like disrupting uPA/uPAR signaling. The review concludes that while pharmacotherapeutic strategies to slow muscle loss through immunosuppression are growing, the rarity of these diseases presents a significant barrier to conducting large-scale clinical trials necessary for FDA approval.