Therapeutic intervention for Niemann–Pick type C disease must target
Descrição
Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect
Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine
Niemann–Pick type C disease: cellular pathology and pharmacotherapy - Wheeler - 2020 - Journal of Neurochemistry - Wiley Online Library
Liposome-targeted recombinant human acid sphingomyelinase: Production, formulation, and in vitro evaluation - ScienceDirect
5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Niemann–Pick type C: a potentially treatable disorder?
Frontiers c-Abl Activation Linked to Autophagy-Lysosomal Dysfunction Contributes to Neurological Impairment in Niemann-Pick Type A Disease
Frontiers Case Report: Be Aware of “New” Features of Niemann–Pick Disease: Insights From Two Pediatric Cases
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