title: Miglustat Does Not Prevent Neurological Involvement in Niemann Pick C Disease.
authors.affiliation: Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa, Italy. Electronic address: majadirocco@ospedale-gaslini.ge.it.
authors.forename: Maja
authors.initials: M
authors.lastname: Di Rocco
authors.affiliation: Pediatric Neurology Policlinico, University of Catania, Catania, Italy.
authors.forename: Rita
authors.initials: R
authors.lastname: Barone
authors.affiliation: Unit of Rare Diseases, Department of Pediatrics, Gaslini Institute, Genoa, Italy.
authors.forename: Annalisa
authors.initials: A
authors.lastname: Madeo
authors.affiliation: Regional Referral Center for Metabolic Diseases, Department of Pediatrics, University of Catania, Catania, Italy.
authors.forename: Agata
authors.initials: A
authors.lastname: Fiumara
mesh_terms: D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives* / Q000627:therapeutic use; D002491:Central Nervous System Agents / Q000627:therapeutic use*; D002648:Child; D003680:Deglutition Disorders / Q000503:physiopathology / Q000517:prevention & control; D005260:Female; D006801:Humans; D008297:Male; D009043:Motor Activity / Q000187:drug effects; D052556:Niemann-Pick Disease, Type C / Q000188:drug therapy* / Q000503:physiopathology*; D012438:Saccades / Q000187:drug effects; D016896:Treatment Outcome
chemical_list: D002491:Central Nervous System Agents; D017485:1-Deoxynojirimycin; C059896:miglustat