ArticleClinical lipidology2012
Therapeutic potential of cyclodextrins in the treatment of Niemann-Pick type C disease.
Article in Clinical lipidology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
36 citing papers in PubMed, 62 citations in OpenAlex.
- An Australian standard of care for Niemann-Pick disease type C.Internal medicine journal · 2026Article
- Generation and characterization of human iPSC-derivedbioRxiv : the preprint server for biology · 2026Article
- Lipofuscin accumulation in aging and neurodegeneration: a potential "timebomb" overlooked in Alzheimer's disease.Translational neurodegeneration · 2025Review
- Drug-Induced Reversible Lysosomal Changes Tracked in Live Cells by Holo-Tomographic Flow Cytometry.ACS nano · 2025Article
- Host-guest systems for the SAMPL9 blinded prediction challenge: phenothiazine as a privileged scaffold for binding to cyclodextrins.Physical chemistry chemical physics : PCCP · 2024Article
- Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.Molecular genetics and metabolism reports · 2023Article
- A differential proteomics study of cerebrospinal fluid from individuals with Niemann-Pick disease, Type C1.Proteomics · 2023Article
- The buzz in the field: the interaction between viruses, mosquitoes, and metabolism.Frontiers in cellular and infection microbiology · 2023Review
- Organ Weights inInternational journal of molecular sciences · 2022Article
- Impaired Retromer Function in Niemann-Pick Type C Disease Is Dependent on Intracellular Cholesterol Accumulation.International journal of molecular sciences · 2021Article
- Article
- Applications of Macrocyclic Host Molecules in Immune Modulation and Therapeutic Delivery.Frontiers in chemistry · 2021Review
- Cyclodextrin-Appended Superparamagnetic Iron Oxide Nanoparticles as Cholesterol-Mopping Agents.Frontiers in chemistry · 2021Article
- Delineation of metabolic responses of Npc1Steroids · 2020Article
- Beyond the Typical Syndrome: Understanding Non-motor Features in Niemann-Pick Type C Disease.Cerebellum (London, England) · 2020Review
- β-Cyclodextrin counteracts obesity in Western diet-fed mice but elicits a nephrotoxic effect.Scientific reports · 2019Article
- Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management.CNS drugs · 2019Review
- Niemann-Pick C1-deficient mice lacking sterol O-acyltransferase 2 have less hepatic cholesterol entrapment and improved liver function.American journal of physiology. Gastrointestinal and liver physiology · 2018Article
- The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity.Acta neuropathologica communications · 2018Article
- Crystal structure of the inclusion complex of cholesterol in β-cyclodextrin and molecular dynamics studies.Beilstein journal of organic chemistry · 2018Article
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
Niemann-Pick type C (NPC) disease is an autosomal recessive neurovisceral lipid and storage disorder characterized by abnormal sequestration of unesterified cholesterol within the late endosomal/lysosomal compartment of all cells in the body. This disease primarily affects children and is characterized by hepatic and pulmonary dysfunction, neurodegeneration and death at an early age. Currently, there is no effective treatment for NPC disease. It was recently discovered that 2-hydroxypropyl-β-cyclodextrin (2HPBCD), when administered systemically to a murine model of either NPC1 or NPC2 disease, significantly reduced lysosomal cholesterol accumulation in almost every organ, delayed the progression of neurodegeneration and significantly prolonged lifespan by allowing trapped cholesterol within the late endosome/lysosome to be released. When 2HPBCD was administered directly into the CNS of
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.