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Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice

Molecular PharmacologyBiochemistryMedicineExperimental TherapeuticNeuropharmacologyPharmacological AgentExperimental PharmacologyPharmacotherapyIminosugar-based InhibitorsBrain Glucosylceramide LevelsPharmacologyInhibitory ActivityDrug DiscoveryNiemann–pick C Mice