Correcting <scp>N</scp>euromuscular <scp>D</scp>eficits <scp>W</scp>ith <scp>G</scp>ene <scp>T</scp>herapy in <scp>P</scp>ompe <scp>D</scp>isease

Abstract

Our data provide new mechanistic insight into the pathology of Pompe disease and suggest that early systemic correction to both neural and muscle tissues may be essential for successful correction of neuromuscular function in Pompe disease. Ann Neurol 2015;78:222-234.