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  2. Guettsches, Anne-Katrin; Meyer, Nancy; Zahedi, René P.; Evangelista, Teresinha; Muentefering, Thomas; Ruck, Tobias; Lacene, Emmanuelle; Heute, Christoph; Gonczarowska-Jorge, Humberto; Schoser, Benedikt; Krause, Sabine; Hentschel, Andreas; Vorgerd, Matthias; Roos, Andreas

    FYCO1 Increase and Effect of Arimoclomol–Treatment in Human VCP–Pathology

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    MDPI AG, 2022

    Published in: Biomedicines

  4. Fog, Cathrine K.; Zago, Paola; Malini, Erika; Solanko, Lukasz M.; Peruzzo, Paolo; Bornaes, Claus; Magnoni, Raffaella; Mehmedbasic, Arnela; Petersen, Nikolaj H.T.; Bembi, Bruno; Aerts, Johannes F.M.G.; Dardis, Andrea; Kirkegaard, Thomas

    The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase

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    Elsevier BV, 2018

    Published in: eBioMedicine

  7. Parfitt, D A; Aguila, M; McCulley, C H; Bevilacqua, D; Mendes, H F; Athanasiou, D; Novoselov, S S; Kanuga, N; Munro, P M; Coffey, P J; Kalmar, B; Greensmith, L; Cheetham, M E

    The heat-shock response co-inducer arimoclomol protects against retinal degeneration in rhodopsin retinitis pigmentosa

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    Springer Science and Business Media LLC, 2014

    Published in: Cell Death & Disease

  10. Machado, Pedro; Barohn, Richard; McDermott, Michael; Blaetter, Thomas; Lloyd, Thomas; Shaibani, Aziz; Freimer, Miriam; Amato, Anthony; Ciafaloni, Emma; Jones, Sarah; Mozaffar, Tahseen; Gibson, Summer; Wicklund, Matthew; Levine, Todd; Sundgreen, Claus; Carstensen, Thomas; Bonefeld, Karen; J⊘rgensen, Anders N⊘rkær; Phonekeo, Karina; Heim, Andrew; Herbelin, Laura; Hanna, Michael; Dimachkie, Mazen

    A Randomized, Double-Blind, Placebo-Controlled Study of Arimoclomol in Patients with Inclusion Body Myositis (S23.010)

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    Ovid Technologies (Wolters Kluwer Health), 2022

    Published in: Neurology

  13. Dimachkie, Mazen; Machado, Pedro; He, Jianghua; Wang, Yunxia; McVey, April; Pasnoor, Mamatha; Gallagher, Philip; Herbelin, Laura; Statland, Jeffrey; Miller, Adrian; Greensmith, Linda; Holton, Janice; Hanna, Michael; Barohn, Richard

    Lack of Correlation Between Biomarker and Clinical Outcome Measures in the Arimoclomol Inclusion Body Myositis Pilot Study (S16.001)

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    Ovid Technologies (Wolters Kluwer Health), 2014

    Published in: Neurology

  15. Fernández Comaduran, Mario; Minotti, Sandra; Jacob-Tomas, Suleima; Rizwan, Javeria; Larochelle, Nancy; Robitaille, Richard; Sephton, Chantelle F.; Vera, Maria; Nalbantoglu, Josephine N.; Durham, Heather D.

    Impact of histone deacetylase inhibition and arimoclomol on heat shock protein expression and disease biomarkers in primary culture models of familial ALS

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    Elsevier BV, 2024

    Published in: Cell Stress and Chaperones

  16. Mengel, Eugen; Patterson, Marc C.; Da Riol, Rosalia M.; Del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Grønborg, Sabine; Harmatz, Paul; Héron, Bénédicte; Maier, Esther M.; Roubertie, Agathe; Santra, Saikat; Tylki‐Szymanska, Anna; Day, Simon; Andreasen, Anne Katrine; Geist, Marie Aavang; Havnsøe Torp Petersen, Nikolaj; Ingemann, Linda; Hansen, Thomas; Blaettler, Thomas; Kirkegaard, Thomas; í Dali, Christine

    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 treatment

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    Wiley, 2021

    Published in: Journal of Inherited Metabolic Disease

  17. Patterson, Marc C.; Mengel, Eugen; Da Riol, Rosalia M.; Del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Grønborg, Sabine; Harmatz, Paul; Héron, Bénédicte; Maier, Esther M.; Roubertie, Agathe; Santra, Saikat; Tylki-Szymańska, Anna; Day, Simon; Andreasen, Anne Katrine; Geist, Marie Aavang; Petersen, Nikolaj Havnsøe Torp; Ingemann, Linda; Hansen, Thomas; Blaettler, Thomas; Kirkegaard, Thomas; Dali, Christine Í.

    Persistent Effect of Arimoclomol in Patients with Niemann-Pick Disease Type C: 24-Month Results from an Open-Label Extension of a Pivotal Phase 2/3 Study

    Conference Proceedings
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    Georg Thieme Verlag KG, 2021

    Published in: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics

  18. Kuta, Rachel; Larochelle, Nancy; Fernandez, Mario; Pal, Arun; Minotti, Sandra; Tibshirani, Michael; St. Louis, Kyle; Gentil, Benoit J.; Nalbantoglu, Josephine N.; Hermann, Andreas; Durham, Heather D.

    Depending on the stress, histone deacetylase inhibitors act as heat shock protein co-inducers in motor neurons and potentiate arimoclomol, exerting neuroprotection through multiple mechanisms in ALS models

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    Elsevier BV, 2020

    Published in: Cell Stress and Chaperones

  19. Machado, P.; Miller, A.; Herbelin, L.; He, J.; Noel, J.; Wang, Y.; McVey, A. L.; Pasnoor, M.; Gallagher, P.; Statland, J.; Brady, S.; Lu, C.-H.; Kalmar, B.; Sethi, H.; Samandouras, G.; Holton, J.; Greensmith, L.; Barohn, R. J.; Hanna, M. G.; Dimachkie, M. M.

    LB0002 Safety and Tolerability of Arimoclomol in Patients with Sporadic Inclusion Body Myositis: A Randomised, Double-Blind, Placebo-Controlled, Phase IIa Proof-of-Concept Trial

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    BMJ, 2013

    Published in: Annals of the Rheumatic Diseases