Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

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Alterations in Proteostasis Mechanisms in Niemann–Pick Type C Disease

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Drug development: Through the barrier

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Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1

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Quantitative Comparison of HSF1 Activators

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Extracellular Hsp90α stimulates a unique innate gene profile in microglial cells with simultaneous activation of Nrf2 and protection from oxidative stress

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Cholesterol-Depletion-Induced Membrane Repair Carries a Raft Conformer of P-Glycoprotein to the Cell Surface, Indicating Enhanced Cholesterol Trafficking in MDR Cells, Which Makes Them Resistant to Cholesterol Modifications

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GEX1A, a Polyketide from Streptomyces chromofuscus, Corrects the Cellular Defects Associated with Niemann-Pick Type C1 in Human Fibroblasts

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Pre-mRNA splicing defects and RNA binding protein involvement in Niemann Pick type C disease

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Beyond the Typical Syndrome: Understanding Non-motor Features in Niemann-Pick Type C Disease

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Harnessing Drug Repurposing for Exploration of New Diseases: An Insight to Strategies and Case Studies

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Movement Disorders and Inherited Metabolic Disorders

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Pharmacological Regulation of Heat Shock Response via Aptamer–Antidote Couple

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Episodic pain in Fabry disease is mediated by a heat shock protein-transient receptor potential cation channel A1 axis

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Development of precision therapies for rare inborn errors of metabolism: Functional investigations in cell culture models

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Regioselective and enantiospecific synthesis of the HSP co-inducer arimoclomol from chiral glycidyl derivatives

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Syntheses and Biological Importance of Herboxidiene/GEX1A

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Niemann-Pick Disease Type C

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Ten plus one challenges in diseases of the lysosomal system

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Abnormal Vertical Eye Movements as a Clue for Diagnosis of Niemann–Pick Type C

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The role of molecular chaperones in the mechanisms of epileptogenesis

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Presenilin Deficiency Results in Cellular Cholesterol Accumulation by Impairment of Protein Glycosylation and NPC1 Function

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A Mouse Systems Genetics Approach Reveals Common and Uncommon Genetic Modifiers of Hepatic Lysosomal Enzyme Activities and Glycosphingolipids

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Correction of Niemann-Pick type C1 disease with the histone deacetylase inhibitor valproic acidPreprint

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Dysregulation of the NLRP3 Inflammasome and Promotion of Disease by IL-1β in a Murine Model of Sandhoff Disease

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Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies

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Interaction with ERp57 is required for progranulin protection against Type 2 Gaucher disease

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Functional Characterization of a Spectrum of Genetic Variants in a Family with Succinic Semialdehyde Dehydrogenase Deficiency

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Intranasal Administration of Hsp70: Molecular and Therapeutic Consequences

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Acetyl-Leucine slows disease progression in lysosomal storage disordersPreprint

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HSP70 reverses lysosomal pathology

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FDA approves Miplyffa and Aqneursa: a new era in treatment of Niemann-Pick Disease Type C

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Experimental pharmacology: Targeting metabolic pathways

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Heat Shock Proteins in Cardiovascular Diseases: From Bench to Bedside

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Arimoclomol as a potential therapy for neuronopathic Gaucher DiseasePreprint

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Amplifying the heat shock response ameliorates pathology in mouse and human models of ALS and FTD.Preprint

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Episodic pain in Fabry disease is mediated by a heat shock protein-TRPA1 axisPreprint

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Entwicklung neuer Therapien für seltene Erkrankungen

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C syndrome - what do we know and what could the future hold?

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Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrinPreprint

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WITHDRAWN: Membrane-lipid therapy: A historical perspective of membrane-targeted therapies—From lipid bilayer structure to the pathophysiological regulation of cells

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Metabolic Treatments of Cerebellar Ataxia

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Genotype-Phenotype Correlation in Noonan Syndrome – Focus on LZTR1 and its Substrates

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TAF1-dependent transcriptional dysregulation underlies multiple sclerosisPreprint

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The Role of Inborn Errors of Metabolism in Intellectual Disability

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The Niemann–Pick diseases

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Therapeutic targeting of neuroinflammation in sphingolipidosis

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Mitochondrial Dysfunction, Endoplasmic Reticulum Stress, and Their Role in Neurodegeneration

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Additional file 1 of Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1

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Additional file 3 of Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1

Campbell, K. S., Cawley, N. X., Luke, R., Scott, K. E. J., Johnson, N., Farhat, N. Y., Alexander, D. M., Wassif, C. A., Li, W., Cologna, S. M., et al. (2023) · Figshare · doi:10.6084/m9.figshare.22607972

Additional file 2 of Identification of cerebral spinal fluid protein biomarkers in Niemann-Pick disease, type C1

Campbell, K. S., Cawley, N. X., Luke, R., Scott, K. E. J., Johnson, N., Farhat, N. Y., Alexander, D. M., Wassif, C. A., Li, W., Cologna, S. M., et al. (2023) · Figshare · doi:10.6084/m9.figshare.22607969

Heat shock protein 70s are modifiers of endothelial function in Fabry disease

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Small Molecules to Elevate Rab7-GTPase Activity and Lower Cholesterol Accumulation in Niemann-Pick Type C Disease

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Elucidating the Vulnerability of Glioblastoma Stem-like Cells to Lysosomal Dysfunctions

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Reply to Chen and Yeh

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Current and emerging therapeutic advancements for Niemann-Pick disease type C: where are we now and what does the future hold?

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Protein aggregation in progressive myoclonus epilepsies and related syndromes

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Effects of bromodomain and extraterminal domain protein inhibition in a mouse model of Niemann-Pick type C diseasePreprint

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Small molecular chaperones regulate protein quality control in neurodegenerative diseases

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Recombinant rat heat shock protein 70 (rrHSP70) may promote remyelination by regulating microglia/macrophages and oligodendrocytes in a toxin-induced demyelination animal model

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HSP70 reverses lysosomal pathology

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Hsp70 stabilizes lysosomes and reverts Niemann–Pick disease-associated lysosomal pathology

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