| Issue Date | Title | Author(s) | Relation | scopus | WOS | Fulltext/Archive link |
| 2006 | Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease | Kishnani, P. S.; Nicolino, M.; Voit, T.; Rogers, R. C.; Tsai, A. C.; Waterson, J.; Herman, G. E.; Amalfitano, A.; Thurberg, B. L.; Richards, S.; Davison, M.; Corzo, D.; Chen, Y. T. | J Pediatr 149(1), 89-97 | | | |
| 1999 | Dubowitz syndrome: a defect in the cholesterol biosynthetic pathway? | Ahmad, A.; Amalfitano, A.; Chen, Y. T.; Kishnani, P. S.; Miller, C.; Kelley, R. | Am J Med Genet 86, 503-504 | | | |
| 2002 | Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction | Ding, E.; Hu, H.; Hodges, B. L.; Migone, F.; Serra, D.; Xu, F.; Chen, Y. T.; Amalfitano, A. | Mol Ther 5, 436-446 | | | |
| 2005 | Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease | An, Y.; Young, S. P.; Kishnani, P. S.; Millington, D. S.; Amalfitano, A.; Corz, D.; Chen, Y. T. | Mol Genet Metab 85, 247-254 | | | |
| 2003 | Long-term correction of glycogen storage disease type II with a hybrid Ad-AAV vector | Sun, B. D.; Chen, Y. T.; Bird, A.; Amalfitano, A.; Koeberl, D. D. | Mol Ther 7, 193-201 | | | |
| 2001 | Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice | Ding, E. Y.; Hodges, B. L.; Hu, H.; McVie-Wylie, A. J.; Serra, D.; Migone, F. K.; Pressley, D.; Chen, Y. T.; Amalfitano, A. | Hum Gene Ther 12, 955-965 | | | |
| 2003 | Multiple muscles in the AMD quail can be ""cross-corrected"" of pathologic glycogen accumulation after intravenous injection of an [E1-, polymerase-] adenovirus vector encoding human acid-alpha-glucosidase | McVie-Wylie, A. J.; Ding, E. Y.; Lawson, T.; Serra, D.; Migone, F. K.; Pressley, D.; Mizutani, M.; Kikuchi, T.; Chen, Y. T.; Amalfitano, A. | J Gene Med 5, 399-406 | | | |
| 2004 | Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease | Hunley, T. E.; Corzo, D.; Dudek, M.; Kishnani, P.; Amalfitano, A.; Chen, Y. T.; Richards, S. M.; Phillips, J. A.; Fogo, A. B.; Tiller, G. E. | Pediatrics 114, e532-e535 | | | |
| 2003 | Packaging of an AAV vector encoding human acid alpha-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector | Sun, B. D.; Chen, Y. T.; Bird, A.; Xu, F.; Hou, Y. X.; Amalfitano, A.; Koeberl, D. D. | Mol Ther 7, 467-477 | | | |
| 2001 | Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial | Amalfitano, A.; Bengur, A. R.; Morse, R. P.; Majure, J. M.; Case, L. E.; Veerling, D. L.; Mackey, J.; Kishnani, P.; Smith, W.; McVie-Wylie, A.; Sullivan, J. A.; Hoganson, G. E.; Phillips, J. A.; Schaefer, G. B.; Charrow, J.; Ware, R. E.; Bossen, E. H.; Chen, Y. T. | Genet Med 3, 132-138 | | | |
| 1999 | Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase | Amalfitano, A.; McVie-Wylie, A. J.; Hu, H.; Dawson, T. L.; Raben, N.; Plotz, P.; Chen, Y. T. | Proc Natl Acad Sci U S A 96, 8861-8866 | | | |
| 2000 | Towards a molecular therapy for glycogen storage disease type II (Pompe disease) | Chen, Y. T.; Amalfitano, A. | Mol Med Today 6, 245-251 | | | |
