Orphazyme (Denmark)

Recent papers from Orphazyme (Denmark)

1. Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C diseaseMolecular Genetics and Metabolism · 2025
2. Measurement properties of the Inclusion Body Myositis Functional Rating ScaleJournal of Neurology Neurosurgery & Psychiatry · 2024
3. Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trialThe Lancet Neurology · 2023
4. Study to Assess Content Validity and Interrater and Intrarater Reliability of the Inclusion Body Myositis Functional Rating ScaleNeurology Clinical Practice · 2023
5. General transcription factor TAF4 antagonizes epigenetic silencing by Polycomb to maintain intestine stem cell functionsCell Death and Differentiation · 2023
6. Heat shock protein amplification improves cerebellar myelination in the Npc1nih mouse modelEBioMedicine · 2022
7. Galactosyl- and glucosylsphingosine induce lysosomal membrane permeabilization and cell death in cancer cellsPLoS ONE · 2022
8. Conformational dynamics of free and membrane-bound human Hsp70 in model cytosolic and endo-lysosomal environmentsCommunications Biology · 2021
9. Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatmentJournal of Inherited Metabolic Disease · 2021
10. Single cell transcriptomics reveal trans-differentiation of pancreatic beta cells following inactivation of the TFIID subunit Taf4Cell Death and Disease · 2021
11. Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United StatesMolecular Genetics and Metabolism · 2021
12. Validation of the 5-domain Niemann-Pick type C Clinical Severity ScaleOrphanet Journal of Rare Diseases · 2021
13. Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort studyOrphanet Journal of Rare Diseases · 2020
14. Clinical disease progression and biomarkers in Niemann–Pick disease type C: a prospective cohort study2020
15. An amphipathic peptide with antibiotic activity against multidrug-resistant Gram-negative bacteriaNature Communications · 2020
16. Clinical Disease Progression and Biomarkers in Niemann–Pick Disease Type C: A Prospective Cohort Study2020
17. HSP70 induces liver X receptor pathway activation and cholesterol reduction in vitro and in vivoTyöväentutkimus Vuosikirja · 2019
18. Animal models for Niemann-Pick type C: implications for drug discovery & developmentExpert Opinion on Drug Discovery · 2019
19. Hsp70 interactions with membrane lipids regulate cellular functions in health and diseaseProgress in Lipid Research · 2019
20. The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidaseEBioMedicine · 2018