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Article

Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder.

Scientific reports - 22 Jun 2018

Kulkarni Aditya, Caporali Paola, Dolas Atul, Johny Soniya, Goyal Sandeep, Dragotto Jessica, Macone Alberto, Jayaraman Ramesh, Fiorenza Maria Teresa

Abstract excerpt

Niemann-Pick Type C1 disorder (NPC) is a rare lysosomal storage disease characterized by the accumulation of cholesterol in lysosomes. NPC has no FDA approved treatments yet, however 2-hydroxypropyl-β-cyclodextrin (HPβCD) has shown efficacy for treating the disease in both mouse and feline NPC models and is currently being investigated in late stage clinical trials. Despite promising results, therapeutic use of...

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Identifiers

PMID: 29934581
DOI: 10.1038/s41598-018-27926-9
PMCID: PMC6015065

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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