Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infant...
Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders
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England: BioMed Central Ltd
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English
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England: BioMed Central Ltd
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Neuronal Ceroid Lipofuscinosis (NCL), also known as Batten disease, is a group of genetically distinct lysosomal disorders that mainly affect the central nervous system, resulting in progressive motor and cognitive decline primarily in children. Multiple distinct genes involved in the metabolism of lipids have been identified to date with various m...
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Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders
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TN_cdi_doaj_primary_oai_doaj_org_article_e67ddb93f0df4fe1a7008a5e8aa42403
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_e67ddb93f0df4fe1a7008a5e8aa42403
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-017-0663-8
