… for enzyme replacement therapy (ERT) for Pompe disease and … skeletal muscle resistance
to therapy in Pompe disease led to … pathophysiology of muscle damage; autophagic defect is …

… enzyme replacement therapy (ERT) for Pompe disease as well as other LSDs since the
recombinant enzymes … Our studies pointed to the role of glycogen autophagy in skeletal muscle …

… of autophagy to the pathophysiology of Pompe disease. … the regenerative capacity of skeletal
muscle in Pompe disease. … , and the inability of the therapeutic enzyme to cross BBB, make …

… autophagy—have become the foundation of our understanding of … of large pools of autophagic
debris in skeletal muscle and the … of using recombinant enzyme to treat skeletal muscle. …

… may yield novel therapeutic targets for these disorders. … ubiquitin ligase like conjugating
enzyme autophagy-related 10 (… defective autophagic pathway in patients with Pompe Disease …

… Currently, the only approved treatment for Pompe disease is enzyme replacement therapy
(ERT… Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease. …

… Autophagic flux refers to the overall rate of autophagic degradation activity: The flux is complete
when the formation of autophagosomes is … LAMP-2A mistargeting and improve oxidative …

… in treating skeletal muscle in Pompe disease are now well recognized, and new therapeutic
… reversal of the autophagic defect in the muscles affected by Pompe disease remains to be …

… of autophagy was a consequence of the enzymatic … skeletal muscle; the therapy relies on
the early restoration of GAA activity, which would be related to the accumulation of autophagy …

… of the autophagic-lysosomal pathway in Pompe disease results into … recombinant enzyme
used for enzyme replacement therapy … skeletal muscles, appear to be relatively refractory to …