Disclosure: P.S.K. and D.S.B. have received research/grant support and honoraria from Genzyme Corporation. P.S.K. is a member of the Pompe and Gaucher Disease Registry Advisory Board for Genzyme Corporation. Recombinant human GAA (rhGAA), in the form of Genzyme's product alglucosidase alfa (Myozyme/Lumizyme) has been approved by the U.S. FDA and the European Union as therapy for Pompe disease. Duke University and those who invented this method of treatment and predecessors of the cell lines used to generate the enzyme (rhGAA) receive royalty payments pursuant to the University's Policy on Inventions, Patents and Technology Transfer.
Molecular analysis and protein processing in late-onset pompe disease patients with low levels of acid α-glucosidase activity
Article first published online: 11 APR 2011
Copyright © 2010 Wiley Periodicals, Inc.
Muscle & Nerve
Volume 43, Issue 5, pages 665–670, May 2011
How to Cite
Bali, D. S., Tolun, A. A., Goldstein, J. L., Dai, J. and Kishnani, P. S. (2011), Molecular analysis and protein processing in late-onset pompe disease patients with low levels of acid α-glucosidase activity. Muscle Nerve, 43: 665–670. doi: 10.1002/mus.21933
- Issue published online: 11 APR 2011
- Article first published online: 11 APR 2011
- Manuscript Accepted: 4 OCT 2010
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