Miglustat: a review of its use in Niemann-Pick disease type C.
about
Niemann-Pick disease treatment: a systematic review of clinical trialsMiglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC DiseaseRecent advances in the diagnosis and treatment of niemann-pick disease type C in children: a guide to early diagnosis for the general pediatricianOrphan drugs: expensive yet necessary.Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease.The development and use of small molecule inhibitors of glycosphingolipid metabolism for lysosomal storage diseases.Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrinPossible FDA-approved drugs to treat Ebola virus infectionA rare case of Niemann-Pick disease type C without neurological involvement in a 66-year-old patient.Sphingolipid lysosomal storage disorders.Dystonia in children and adolescents: a systematic review and a new diagnostic algorithm.Treatable causes of cerebellar ataxia.A novel diagnostic approach to patients with myoclonus.Small molecules as therapeutic agents for inborn errors of metabolism.Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann-Pick type C disease: review of initial published cases.Pearls & Oy-sters: Niemann-Pick disease type C in a 65-year-old patientCyclodextrin Alters GABAergic Input to CA1 Pyramidal Cells in Wild-Type But Not in NPC1-Deficient Mice.Determination of protonation states of iminosugar-enzyme complexes using photoinduced electron transfer.High-content screen for modifiers of Niemann-Pick Type C disease in patient cells.Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice.Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder.Abnormal Vertical Eye Movements as a Clue for Diagnosis of Niemann-Pick Type C.Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials
P2860
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P2860
Miglustat: a review of its use in Niemann-Pick disease type C.
description
2014 nî lūn-bûn
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2014 թուականի Յունուարին հրատարակուած գիտական յօդուած
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2014 թվականի հունվարին հրատարակված գիտական հոդված
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2014年の論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年论文
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name
Miglustat: a review of its use in Niemann-Pick disease type C.
@ast
Miglustat: a review of its use in Niemann-Pick disease type C.
@en
type
label
Miglustat: a review of its use in Niemann-Pick disease type C.
@ast
Miglustat: a review of its use in Niemann-Pick disease type C.
@en
prefLabel
Miglustat: a review of its use in Niemann-Pick disease type C.
@ast
Miglustat: a review of its use in Niemann-Pick disease type C.
@en
P2860
P1433
P1476
Miglustat: a review of its use in Niemann-Pick disease type C.
@en
P2860
P2888
P356
10.1007/S40265-013-0164-6
P577
2014-01-01T00:00:00Z
P6179
1005473762