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

• Published in: Neurological sciences Vol. 38; no. 5; pp. 727 - 743
• Main Authors: 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: Journal Article
• Language: English
• Published: Milan Springer Milan 01.05.2017; Springer Nature B.V
• Subjects: 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; 2-Hydroxypropyl-β-cyclodextrin; Effectiveness; Toxicity; Niemann–Pick

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...