Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
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Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersNiemann-Pick disease treatment: a systematic review of clinical trialsNiemann-Pick disease type C symptomatology: an expert-based clinical descriptionRecent advances in the diagnosis and treatment of niemann-pick disease type C in children: a guide to early diagnosis for the general pediatricianLong term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type CMiglustat: a review of its use in Niemann-Pick disease type C.Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells.Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrinDevelopment of targeted therapies for Parkinson's disease and related synucleinopathiesStable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.Disease and patient characteristics in NP-C patients: findings from an international disease registry.Normalisation of brain spectroscopy findings in Niemann-Pick disease type C patients treated with miglustat.Niemann-Pick type C Suspicion Index tool: analyses by age and association of manifestations.Relative acidic compartment volume as a lysosomal storage disorder-associated biomarkerSphingolipid lysosomal storage disorders.Modeling Niemann Pick type C1 using human embryonic and induced pluripotent stem cells.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.EFNS/ENS Consensus on the diagnosis and management of chronic ataxias in adulthood.Phenotypic heterogeneity of Niemann-Pick disease type C in monozygotic twins.Distinguishing neurocognitive deficits in adult patients with NP-C from early onset Alzheimer's dementia.Miglustat in Niemann-Pick disease type C patients: a review
P2860
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P2860
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年学术文章
@wuu
2012年学术文章
@zh-cn
2012年学术文章
@zh-hans
2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
@yue
2012年學術文章
@zh
2012年學術文章
@zh-hant
name
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@en
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@nl
type
label
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@en
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@nl
prefLabel
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@en
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C.
@nl
P2093
P2860
P50
P1476
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C
@en
P2093
P2860
P2888
P304
P356
10.1007/S10545-012-9479-9
P577
2012-04-05T00:00:00Z
P5875
P6179
1006601850