Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C-Reference-Cited by-同舟云学术

Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C

Published:2012-04-05 Issue:1 Volume:36 Page:129-137
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

Chien Y. H.,Peng S. F.,Yang C. C.,Lee N. C.,Tsai L. K.,Huang A. C.,Su S. C.,Tseng C. C.,Hwu W. L.

Publisher

Wiley

Subject

Genetics(clinical),Genetics

Link

http://www.springerlink.com/index/pdf/10.1007/s10545-012-9479-9

Reference27 articles.

1. Actelion (2010) Miglustat (Zavesca®) Summary of product characteristics. Retrieved 4 July 2011, from http://eudrapharm.eu/eudrapharm/showDocument?documentId=299255761182430949

2. Carstea ED, Morris JA, Coleman KG et al (1997) Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis. Science 277:228–231

3. Chien YH, Lee NC, Tsai LK et al (2007) Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year. J Inherit Metab Dis 30:826

4. Fecarotta S, Amitrano M, Romano A et al (2011) The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat. Am J Med Genet A 155A:540–547

5. Han TR, Paik NJ, Park JW (2001) Quantifying swallowing function after stroke: A functional dysphagia scale based on videofluoroscopic studies. Arch Phys Med Rehabil 82:677–682

Cited by 35 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Overview of clinical, molecular, and therapeutic features of Niemann–Pick disease (types A, B, and C): Focus on therapeutic approaches;Cell Biochemistry and Function;2024-05-07

2. Loss of Flot2 expression in deep cerebellar nuclei neurons of mice with Niemann-Pick disease type C;Heliyon;2023-07

3. Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review;European Journal of Neurology;2023-06-08

4. Possible genotype-phenotype correlations in Niemann-Pick type C patients and miglustat treatment;Ideggyógyászati szemle;2021

5. Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1;JAMA Neurology;2020-12-01