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One of these drugs,\n                    <jats:italic>N<\/jats:italic>\n                    \u2013butyldeoxynojirimycin (NB\u2013DNJ), shows efficacy in mouse models of Tay\u2013Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB\u2013DNJ may be of therapeutic benefit, at least in the juvenile and adult onset variants of these disorders. The infantile onset variants will require an additional enzyme\u2013augmenting modality if the pathology is to be significantly improved. A second drug,\n                    <jats:italic>N<\/jats:italic>\n                    \u2013butyldeoxyglactonojirimycin, looks very promising for treating storage diseases with neurological involvement as high systemic dosing is achievable without any side\u2013effects.\n                  <\/jats:p>","DOI":"10.1098\/rstb.2003.1279","type":"journal-article","created":{"date-parts":[[2003,9,9]],"date-time":"2003-09-09T11:27:32Z","timestamp":1063106852000},"page":"947-954","source":"Crossref","is-referenced-by-count":49,"title":["Substrate reduction therapy in mouse models of the glycosphingolipidoses"],"prefix":"10.1098","volume":"358","author":[{"given":"Frances M.","family":"Platt","sequence":"first","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mylvaganam","family":"Jeyakumar","sequence":"additional","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ulrika","family":"Andersson","sequence":"additional","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tanya","family":"Heare","sequence":"additional","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Raymond A.","family":"Dwek","sequence":"additional","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terry D.","family":"Butters","sequence":"additional","affiliation":[{"name":"Glycobiology Institute, Department of Biochemistry, University of Oxford, South Parks Road, Oxford OX1 3QU, UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"175","published-online":{"date-parts":[[2003,5,29]]},"reference":[{"key":"p_1","unstructured":"Tay-Sachs Yamanaka et al. 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