Targeted Disruption of the Acid α-Glucosidase Gene in Mice Causes an Illness with Critical Features of Both Infantile and Adult Human Glycogen Storage Disease Type IINina Raben, Paul H. Plötz, Kanneboyina Nagaraju et al.|Journal of Biological Chemistry|1998Cited by 298
Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe diseaseMarc Nicolino, Priya S. Kishnani, Barry J. Byrne et al.|Genetics in Medicine|2009Cited by 292
Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2DJerry R. Mendell, K. Reed Clark, Louise R. Rodino‐Klapac et al.|Annals of Neurology|2010Cited by 243
Acid a-Glucosidase Deficiency (Glycogenosis Type II, Pompe Disease)Nina Raben, Barry J. Byrne, Paul H. Plötz|Current Molecular Medicine|2002Cited by 235
Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteinsJerry R. Mendell, K. Reed Clark, Janaiah Kota et al.|Annals of Neurology|2009Cited by 214