Therapeutic intervention for Niemann–Pick type C disease must target
Por um escritor misterioso
Last updated 07 abril 2025


Protocol for initiation of miglustat treatment in NP-C. Reproduced with

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine

PDF) Recent Advances in the Diagnosis and Treatment of Niemann-Pick Disease Type C in Children: A Guide to Early Diagnosis for the General Pediatrician

Targeting defective sphingosine kinase 1 in Niemann–Pick type C disease with an activator mitigates cholesterol accumulation - Journal of Biological Chemistry

IJMS, Free Full-Text

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Main therapies and point of action in Niemann type C disease.

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics

5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
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NNPDF on X: #NNPDF partnered with @RareDiseases to create this animated educational resource on Niemann Pick Type C. Please help us raise awareness of #NPC by sharing this video with your networks07 abril 2025
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