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As orphan drug regulations, however, encouraged development of drugs for these disorders by granting marketing exclusivity for 10 years and other commercial benefits, enzyme replacement therapy became available for lysosomal storage disorders, such as Gaucher disease, Fabry disease, mucopolysaccharidoses type I, II, and VI, and Pompe disease. This review will summarize the efficacy and clinical status of hematopoietic stem cell transplantation, enzyme replacement, and substrate deprivation therapy, and describe new therapeutic perspectives currently under preclinical investigations such as chaperone\u2010mediated therapy, stop\u2010codon read\u2010through therapy, and gene therapy. \u00a9 2009 IUBMB IUBMB Life, 62(1):33\u201340, 2010<\/jats:p>","DOI":"10.1002\/iub.284","type":"journal-article","created":{"date-parts":[[2009,12,14]],"date-time":"2009-12-14T18:36:57Z","timestamp":1260815817000},"page":"33-40","source":"Crossref","is-referenced-by-count":99,"title":["Therapy for lysosomal storage disorders"],"prefix":"10.1002","volume":"62","author":[{"given":"Michael","family":"Beck","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2009,12,14]]},"reference":[{"key":"e_1_2_8_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbamcr.2008.12.001"},{"key":"e_1_2_8_3_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.162.3853.570"},{"key":"e_1_2_8_4_2","doi-asserted-by":"publisher","DOI":"10.1007\/s00281-004-0166-2"},{"key":"e_1_2_8_5_2","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1703839"},{"key":"e_1_2_8_6_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa032613"},{"key":"e_1_2_8_7_2","doi-asserted-by":"publisher","DOI":"10.1097\/00019052-199904000-00007"},{"key":"e_1_2_8_8_2","first-page":"471","article-title":"Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X\u2010adrenoleukodystrophy","volume":"10","author":"Cartier N.","year":"2008","journal-title":"Curr. 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