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Disease Areas
MuscularDystrophy
Muscular Dystrophy (ATC-401, 402, 403)

Duchene muscular dystrophy is an inherited X-linked disease caused by mutations in the dystrophin gene, which leads to sarcolemmal disruption, cytosolic calcium channel activation and progressive muscle wasting[7]. Our autophagy-regulating compounds can inhibit the excessive degradation of actin and other cytoskeletons, thereby halting muscle wasting as a potential cure for Duchene muscular dystrophy.

[7] Agnieszka Łoboda. et. al. Muscle and cardiac therapeutic strategies for Duchenne muscular dystrophy: past, present, and future. Pharmacol Rep.

Sources : https://www.mda.org/