Recombinant human acid α-glucosidase: Major clinical benefits in infantile-onset Pompe disease
P. S. Kishnani, D. Corzo, M. Nicolino, B. Byrne, H. Mandel, W. L. Hwu, N. Leslie, J. Levine, C. Spencer, M. McDonald, J. Li, J. Dumontier, M. Halberthal, Y. H. Chien, R. Hopkin, S. Vijayaraghavan, D. Gruskin, D. Bartholomew, Ans van der Ploeg, J. P. Clancy, R. Parini, G. Morin, M. Beck, G. S. De la Gastine, M. Jokic, B. Thurberg, S. Richards, D. Bali, M. Davison, M Worden, Y. T. Chen, J. E. Wraith
Cited by 9Open Access
Abstract
Background: Pompe disease is a progressive metabolic neuromuscular disorder resulting from deficiency of lysosomal acid -glucosidase (GAA). Infantile-onset Pompe disease is characterized by cardiomyopathy, respiratory and skeletal muscle weakness, and early death. The safety and efficacy of recombinant human (rh) GAA were evaluated in 18 patients with rapidly progressing infantile-onset Pompe disease.
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