Open access
Aug 2026
Axonopathy in Duchenne Muscular Dystrophy limits microdystrophin gene therapy efficacy
DMD is redefined as an integrated motor unit pathology, thereby underscoring the absolute necessity of implementing combined therapeutic strategies that target both the muscle and the peripheral nervous system.
Aly Bourguiba-Villeneuve, Maxime Gelin, Aurélie Fail et al.
· bioRxiv · 0 citations