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Despite improved survival and growth, long\u2010term complications of GSD type I (GSD I) have not responded to dietary therapy with uncooked cornstarch or continuous gastric feeding. The recognized significant risk of renal disease and liver malignancy in GSD I has prompted efforts towards curative therapy, including organ transplantation, in those deemed at risk. Results of clinical trials in infantile Pompe disease with alglucosidase alfa (Myozyme) showed prolonged survival reversal of cardiomyopathy, and motor gains. This resulted in broad label approval of Myozyme for Pompe disease in 2006. Furthermore, the development of experimental therapies, such as adeno\u2010associated virus (AAV) vector\u2010mediated gene therapy, holds promise for the availability of curative therapy in GSD I and GSD II\/Pompe disease in the future.<\/jats:p>","DOI":"10.1007\/s10545-007-0519-9","type":"journal-article","created":{"date-parts":[[2007,2,16]],"date-time":"2007-02-16T13:52:10Z","timestamp":1171633930000},"page":"159-164","source":"Crossref","is-referenced-by-count":59,"title":["Glycogen storage disease types I and II: Treatment updates"],"prefix":"10.1002","volume":"30","author":[{"given":"D. D.","family":"Koeberl","sequence":"first","affiliation":[{"name":"Division of Medical Genetics\/Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P. 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