title: Niemann-Pick disease treatment: a systematic review of clinical trials.
abstract: The aim of this systematic review was to analyse all the published clinical trials assessing treatments for Niemann-Pick (NP) disease. At present there are only trials investigating the treatment of NP disease type C. Furthermore, there is no uniformity among studies in treatment outcomes or in data analysis and presentation of results. Miglustat is able to delay neurodegeneration, with greater benefits in patients with a late onset of the disease and β-cyclodextrin-hydroxypropyl (HBP-CD) can attenuate clinical symptoms. As for cholesterol-lowering drugs, the combination of lovastatin, cholestyramine and nicotinic acid is the most effective one for lowering cholesterolemia. Further research is much needed, and ongoing trials using enzyme replacement therapy might hopefully show promising results in the foreseeable future.
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Alejandro
authors.initials: A
authors.lastname: Santos-Lozano
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Diana
authors.initials: D
authors.lastname: Villamandos García
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Fabian
authors.initials: F
authors.lastname: Sanchis-Gomar
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Carmen
authors.initials: C
authors.lastname: Fiuza-Luces
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Helios
authors.initials: H
authors.lastname: Pareja-Galeano
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Nuria
authors.initials: N
authors.lastname: Garatachea
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Gisela
authors.initials: G
authors.lastname: Nogales Gadea
authors.affiliation: 1 Research Institute of Hospital 12 de Octubre ("i+12"), Madrid, Spain ; 2 GIDFYS, Department of Health Sciences, European University Miguel de Cervantes, Valladolid, Spain ; 3 Faculty of Health Science, University of León, Ponferrada, Spain ; 4 Department of Physiotherapy and Nursing, University of Zaragoza, Huesca, Spain ; 5 GENUD (Growth, Exercise, Nutrition and Development) Research Group, University of Zaragoza, Zaragoza, Spain ; 6 Translational Research Laboratoy in Neuromuscular Diseases, Neurosciences Department, Germans Trias i Pujol Research Institute and Campus Can Ruti, Autonomous University of Barcelona, Badalona, Spain ; 7 European University, Madrid, Spain.
authors.forename: Alejandro
authors.initials: A
authors.lastname: Lucia
keywords: Niemann-Pick (NP) disease; clinical trial; miglustat; treatment