biostudies-literatureUnknown13(1)Pineda M<h4>Objective</h4>Niemann-Pick disease type C (NP-C) is a rare, autosomal recessive, neurodegenerative disease associated with a wide variety of progressive neurological manifestations. Miglustat is indicated for the treatment of progressive neurological manifestations in both adults and children. Since approval in 2009 there has been a vast growth in clinical experience with miglustat. The effectiveness of miglustat has been assessed using a range of measures.<h4>Methods</h4>Comprehensive review of published data from studies of cellular neuropathological markers and structural neurological indices in the brain, clinical impairment/disability, specific clinical neurological manifestations, and patient survival.<h4>Results</h4>Cranial diffusion tensor imaging and magnetic resonance spectroOrphanet journal of rare diseases140https://www.ebi.ac.uk/biostudies/studies/S-EPMC6094874biostudies-literatureMiglustat in Niemann-Pick disease type C patients: a review.PMC6094874Pineda MWalterfang MPatterson MCfalseMiglustat in Niemann-Pick disease type C patients: a review.<h4>Objective</h4>Niemann-Pick disease type C (NP-C) is a rare, autosomal recessive, neurodegenerative disease associated with a wide variety of progressive neurological manifestations. Miglustat is indicated for the treatment of progressive neurological manifestations in both adults and children. Since approval in 2009 there has been a vast growth in clinical experience with miglustat. The effectiveness of miglustat has been assessed using a range of measures.<h4>Methods</h4>Comprehensive review of published data from studies of cellular neuropathological markers and structural neurological indices in the brain, clinical impairment/disability, specific clinical neurological manifestations, and patient survival.<h4>Results</h4>Cranial diffusion tensor imaging and magnetic resonance spectro2018-01-01T00:00:00Z2018 Aug2026-07-16T01:41:35.507Z2019-03-26T23:52:17ZS-EPMC60948743011133410.1186/s13023-018-0844-0