PhysiologybioRxivPreprint
Targeting lysosomal damage is a new therapeutic perspective for Duchenne Muscular Dystrophy
Abbass Jaber, Laura Palmieri, Rania Bakour…·David Israeli
This study identifies lysosomal damage as a novel mechanism in Duchenne Muscular Dystrophy and shows that combining trehalose with gene therapy improves outcomes.
DOI 10.1101/2025.02.11.637610