Structure of human lysosomal acid α-glucosidase–a guide for the treatment of Pompe disease
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Physics and Astronomy,General Biochemistry, Genetics and Molecular Biology,General Chemistry
Link
http://www.nature.com/articles/s41467-017-01263-3.pdf
Reference49 articles.
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2. van der Ploeg, A. T. & Reuser, A. J. Pompe’s disease. Lancet 372, 1342–1353 (2008).
3. Raben, N., Plotz, P. & Byrne, B. J. Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease). Curr. Mol. Med. 2, 145–166 (2002).
4. Parenti, G. & Andria, G. Pompe disease: from new views on pathophysiology to innovative therapeutic strategies. Curr. Pharm. Biotechnol. 12, 902–915 (2011).
5. Van den Hout, J. M. et al. Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk. Pediatrics 113, e448–e457 (2004).
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