Targeted Therapies Aim at the Root of the Disease
At the 2025 Muscular Dystrophy Association Clinical & Scientific Conference in Dallas, Dr. Matthew Alexander, associate professor of pediatric neurology and genetics at the University of Alabama, Birmingham, led a compelling session on the future of muscular dystrophy treatment. The focus? Targeted therapies are designed to target the signaling pathways that drive the disease.
Muscular dystrophies, including Duchenne muscular dystrophy (DMD), are a group of genetic disorders that lead to progressive muscle weakness. Traditional treatments have primarily centered on managing symptoms. Now, researchers are digging deeper into the molecular roots of the disease.
Alexander and fellow experts highlighted how disrupted signaling pathways influence fibrosis (the buildup of scar tissue), inflammation, and calcium imbalance—all major contributors to muscle degeneration. Of particular interest is fibrosis, which affects not just skeletal muscle but also heart and lung tissue. Blocking these fibrotic pathways could lead to significant improvements in overall patient health.
Another key area of focus is stabilizing the muscle cell (myofiber) membranes to prevent calcium leakage and immune overactivation, both of which worsen the disease. However, Alexander cautioned that the path to effective treatment is complex due to genetic variability and patient-specific factors. This makes a personalized approach essential.
These insights point toward a future where treatments not only slow progression but target the fundamental causes of muscular dystrophies.
Read the full article here: Exploring New Avenues for Treating Muscular Dystrophies With Targeted Therapies: Matthew Alexander, PhD by Matthew Alexander, PhD,Marco Meglio., March 25, 2025, NeurologyLive