Miglustat therapy in a case of early-infantile Niemann-Pick type C-Reference-Cited by-同舟云学术

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Miglustat therapy in a case of early-infantile Niemann-Pick type C

Published:2017-11 Issue:10 Volume:39 Page:886-890
ISSN:0387-7604
Container-title:Brain and Development
language:en
Short-container-title:Brain and Development

Author:

Usui Miho,Miyauchi Akihiko,Nakano Yuko,Nakamura Sachie,Jimbo Eriko,Itamura Shinji,Adachi Kaori,Nanba Eiji,Narita Aya,Yamagata Takanori,Osaka Hitoshi

Publisher

Elsevier BV

Subject

Neurology (clinical),Developmental Neuroscience,General Medicine,Pediatrics, Perinatology and Child Health

Reference10 articles.

1. Niemann-Pick disease type C;Vanier;Orphanet J Rare Dis,2010

2. Miglustat: a review of its use in Niemann-Pick disease type C;Lyseng-Williamson;Drugs,2014

3. Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: a case series;Pineda;Mol Genet Metab,2010

4. Early miglustat therapy in infantile Niemann-Pick disease type C;Di Rocco;Pediatr Neurol,2012

5. Effects of miglustat on stabilization of neurological disorder in niemann-pick disease type C: Iranian pediatric case series;Karimzadeh;J Child Neurol,2013

Cited by 7 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. Peripheral neuropathy as a very rare symptom in a patient with Niemann–Pick type C with negative enzymatic evaluation: a case report;Journal of Medical Case Reports;2022-01-12

3. Normal long‐term neurologic and graft outcome after liver transplantation in an infant with Neimann‐Pick type C disease;American Journal of Transplantation;2021-09-12

4. Favorable outcomes following early onset oral miglustat in early infantile Niemann Pick Type C;Molecular Genetics and Metabolism Reports;2021-06

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

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