Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann–Pick type C disease: review of initial published cases

Niemann–Pick type C (NP-C) is a rare neurodegenerative disorder. Management is mainly supportive and symptomatic. The investigational use of 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) showed a promising role in treating NP-C, although efficacy and safety have not been established. We conducted searche...

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Veröffentlicht in:	Neurological sciences 2017-05, Vol.38 (5), p.727-743
Hauptverfasser:	Megías-Vericat, Juan Eduardo, García-Robles, Ana, Company-Albir, María José, Fernández-Megía, María José, Pérez-Miralles, Francisco Carlos, López-Briz, Eduardo, Casanova, Bonaventura, Poveda, José Luis
Format:	Artikel
Sprache:	eng
Schlagworte:	2-Hydroxypropyl-beta-cyclodextrin - therapeutic use Age Biomarkers Brain diseases Cholesterol Clinical trials Community support Databases, Bibliographic - statistics & numerical data Dementia Excipients - therapeutic use FDA approval Genetic disorders Humans Medical libraries Medicine Medicine & Public Health Metabolic disorders Mutation Neurology Neuroradiology Neurosciences Neurosurgery Niemann-Pick Disease, Type C - drug therapy Product development Psychiatry Review Article
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