CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.
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Less Is More: Substrate Reduction Therapy for Lysosomal Storage DisordersGlucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models.Progress and potential of non-inhibitory small molecule chaperones for the treatment of Gaucher disease and its implications for Parkinson disease.N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV.Small molecules as therapeutic agents for inborn errors of metabolism.The Complicated Relationship between Gaucher Disease and Parkinsonism: Insights from a Rare Disease.Modulating ryanodine receptors with dantrolene attenuates neuronopathic phenotype in Gaucher disease mice.Protein misfolding diseases: prospects of pharmacological treatment.The Spectrum of Neurological Manifestations Associated with Gaucher Disease.Current and Future Treatments for Lysosomal Storage Disorders.Oncogenic dependency on β-catenin in liver cancer cell lines correlates with pathway activation.Translational Aspects of Sphingolipid Metabolism in Renal Disorders.A HILIC-MS/MS method for simultaneous quantification of the lysosomal disease markers galactosylsphingosine and glucosylsphingosine in mouse serum
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CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 07 March 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
@en
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
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type
label
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
@en
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
@nl
prefLabel
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
@en
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease.
@nl
P2093
P2860
P356
P1433
P1476
CNS-accessible Inhibitor of Gl ...... Neuronopathic Gaucher Disease
@en
P2093
Amy Allaire
Dinesh S Bangari
Eva Budman
Gregory A Grabowski
Hyejung Park
Jennifer B Nietupski
John P Leonard
Mary A Cromwell
Seng H Cheng
P2860
P304
P356
10.1038/MT.2016.53
P577
2016-03-07T00:00:00Z