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Neurobiology of Disease, ISSN 0969-9961, 2017, Volume 105, pp. 257 - 270
Abstract Mucolipidosis type IV (MLIV) is a lysosomal storage disease exhibiting progressive intellectual disability, motor impairment, and premature death.... 
Neurology | small molecule therapy | mucolipidosis type IV | lysosomal storage disease | Purkinje cells | glycosphingolipids | miglustat | mucolipin-1
Journal Article
Journal Article
Journal Article
Neurobiology of Disease, ISSN 0969-9961, 09/2017, Volume 105, pp. 257 - 270
Journal Article
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2013, Volume 8, Issue 2, p. e56780
Journal Article