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Affected individuals exhibit either severe hypotonia associated with hypertrophic cardiomyopathy (infantile forms) or progressive muscle weakness (late\u2010onset forms). Even if enzyme replacement therapy has recently become a standard treatment, it suffers from several limitations. This review will present the main results of enzyme replacement therapy and the recent findings concerning alternative treatments for Pompe disease, such as gene therapy, enzyme enhancement therapy, and substrate reduction therapy. \u00a9 2011 IUBMB IUBMB Life, 63(11): 979\u2013986, 2011<\/jats:p>","DOI":"10.1002\/iub.529","type":"journal-article","created":{"date-parts":[[2011,10,14]],"date-time":"2011-10-14T13:10:32Z","timestamp":1318597832000},"page":"979-986","source":"Crossref","is-referenced-by-count":17,"title":["New insights into therapeutic options for Pompe 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