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Non\u2010specific therapies such as hematopoietic stem cell transplantation have been useful in globoid cell leukodystrophy (Krabbe disease) and in some mucopolysaccharidoses. Anti\u2010inflammatory agents also show promise as adjuvant therapy. High doses of replacement therapy with native or modified enzyme show renewed promise for correction of CNS cells. Alternatively, small molecules can enter the brain relatively easily and promote reduction of accumulated substrate or function as pharmacological chaperones to enhance the level of the deficient enzyme. Gene therapy is still being developed and tested in patients. It is therefore likely that, thanks to a better understanding of disease mechanism, a variety of therapeutic approaches, used alone or in combination, will be useful to treat the devastating neurological complications of LSDs.<\/jats:p>","DOI":"10.1007\/s10545-010-9047-0","type":"journal-article","created":{"date-parts":[[2010,2,16]],"date-time":"2010-02-16T08:20:53Z","timestamp":1266308453000},"page":"373-379","source":"Crossref","is-referenced-by-count":32,"title":["Therapeutic approaches for neuronopathic lysosomal storage disorders"],"prefix":"10.1002","volume":"33","author":[{"given":"Raphael","family":"Schiffmann","sequence":"first","affiliation":[{"name":"Institute of Metabolic Disease, Baylor Research Institute Dallas TX 75226 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2010,2,17]]},"reference":[{"key":"e_1_2_4_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2009.07.030"},{"key":"e_1_2_4_3_1","doi-asserted-by":"publisher","DOI":"10.1097\/AOG.0b013e3181bd12f4"},{"key":"e_1_2_4_4_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI9711"},{"key":"e_1_2_4_5_1","doi-asserted-by":"publisher","DOI":"10.1096\/fj.07-102715"},{"key":"e_1_2_4_6_1","doi-asserted-by":"publisher","DOI":"10.2174\/138161208784705504"},{"key":"e_1_2_4_7_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI200419205"},{"key":"e_1_2_4_8_1","doi-asserted-by":"publisher","DOI":"10.1203\/00006450-200201000-00009"},{"key":"e_1_2_4_9_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddn237"},{"key":"e_1_2_4_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/bit.21602"},{"key":"e_1_2_4_11_1","doi-asserted-by":"publisher","DOI":"10.1146\/annurev.med.57.110104.115650"},{"key":"e_1_2_4_12_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp.2008.143693"},{"key":"e_1_2_4_13_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.2185-07.2007"},{"key":"e_1_2_4_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm.2025"},{"key":"e_1_2_4_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.32861"},{"key":"e_1_2_4_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2006.12.012"},{"key":"e_1_2_4_17_1","doi-asserted-by":"publisher","DOI":"10.1096\/fasebj.14.2.361"},{"key":"e_1_2_4_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.02.005"},{"key":"e_1_2_4_19_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa042604"},{"key":"e_1_2_4_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1742-4658.2007.06041.x"},{"key":"e_1_2_4_21_1","first-page":"299","article-title":"The use of partially HLA\u2010mismatched donors for allogeneic transplantation in patients with mucopolysaccharidosis\u2010I","volume":"2","author":"Fleming DR","year":"1998","journal-title":"Pediatr Transplant"},{"key":"e_1_2_4_22_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddm223"},{"key":"e_1_2_4_23_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0712147105"},{"key":"e_1_2_4_24_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0803715105"},{"key":"e_1_2_4_25_1","first-page":"157","article-title":"Sanfilippo disease type B. 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