Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 t... von Buyse, Gunnar M, Prof, Voit, Thomas, Prof, Schara, Ulrike, Prof, Straathof, Chiara S M, MD, D'Angelo, M Grazia, MD, Bernert, Günther, MD, Cuisset, Jean-Marie, MD, Finkel, Richard S, Prof, Goemans, Nathalie, MD, McDonald, Craig M, Prof, Rummey, Christian, PhD, Meier, Thomas, PhD

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The neurogenic bladder: medical treatment von Verpoorten, Carla, Buyse, Gunnar M.

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Human iPSC model reveals a central role for NOX4 and oxidative stress in Duchenne cardiomyopathy von Duelen, Robin, Costamagna, Domiziana, Gilbert, Guillaume, De Waele, Liesbeth, Goemans, Nathalie, Desloovere, Kaat, Verfaillie, Catherine M., Sipido, Karin R., Buyse, Gunnar M., Sampaolesi, Maurilio

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Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study von Goemans, Nathalie M, Tulinius, Már, van den Hauwe, Marleen, Kroksmark, Anna-Karin, Buyse, Gunnar, Wilson, Rosamund J, van Deutekom, Judith C, de Kimpe, Sjef J, Lourbakos, Afrodite, Campion, Giles

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy von Goemans, Nathalie M, Tulinius, Mar, van den Akker, Johanna T, Burm, Brigitte E, Ekhart, Peter F, Heuvelmans, Niki, Holling, Tjadine, Janson, Anneke A, Platenburg, Gerard J, Sipkens, Jessica A, Sitsen, J.M. Ad, Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B, Buyse, Gunnar, Darin, Niklas, Verschuuren, Jan J, Campion, Giles V, de Kimpe, Sjef J, van Deutekom, Judith C

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Gain-of-function FHF1 mutation causes early-onset epileptic encephalopathy with cerebellar atrophy von Siekierska, Aleksandra, Isrie, Mala, Liu, Yue, Scheldeman, Chloë, Vanthillo, Niels, Lagae, Lieven, de Witte, Peter A.M, Van Esch, Hilde, Goldfarb, Mitchell, Buyse, Gunnar M

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Long-term data with idebenone on respiratory function outcomes in patients with Duchenne muscular dystrophy von Servais, Laurent, Straathof, Chiara S.M., Schara, Ulrike, Klein, Andrea, Leinonen, Mika, Hasham, Shabir, Meier, Thomas, De Waele, Liesbeth, Gordish-Dressman, Heather, McDonald, Craig M., Mayer, Oscar H., Voit, Thomas, Mercuri, Eugenio, Buyse, Gunnar M.

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Defining the phenotype of FHF1 developmental and epileptic encephalopathy von Trivisano, Marina, Ferretti, Alessandro, Bebin, Elizabeth, Huh, Linda, Lesca, Gaetan, Siekierska, Aleksandra, Takeguchi, Ryo, Carneiro, Maryline, De Palma, Luca, Guella, Ilaria, Haginoya, Kazuhiro, Shi, Ruo Ming, Kikuchi, Atsuo, Kobayashi, Tomoko, Jung, Julien, Lagae, Lieven, Milh, Mathieu, Mathieu, Marie L., Minassian, Berge A., Novelli, Antonio, Pietrafusa, Nicola, Takeshita, Eri, Tartaglia, Marco, Terracciano, Alessandra, Thompson, Michelle L., Cooper, Gregory M., Vigevano, Federico, Villard, Laurent, Villeneuve, Nathalie, Buyse, Gunnar M., Demos, Michelle, Scheffer, Ingrid E., Specchio, Nicola

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Effects of glucocorticoids and idebenone on respiratory function in patients with duchenne muscular dystrophy von Buyse, Gunnar M., Goemans, Nathalie, van den Hauwe, Marleen, Meier, Thomas

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Extracellular vesicle-derived miRNAs improve stem cell-based therapeutic approaches in muscle wasting conditions von Yedigaryan, Laura, Martínez-Sarrà, Ester, Giacomazzi, Giorgia, Giarratana, Nefele, van der Veer, Bernard K, Rotini, Alessio, Querceto, Silvia, Grosemans, Hanne, Cortés-Calabuig, Álvaro, Salucci, Sara, Battistelli, Michela, Falcieri, Elisabetta, Gijsbers, Rik, Quattrocelli, Mattia, Peng Koh, Kian, De Waele, Liesbeth, Buyse, Gunnar M, Derua, Rita, Sampaolesi, Maurilio

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The contribution of platelet studies to the understanding of disease mechanisms in complex and monogenetic neurological disorders von Goubau, Christophe, Buyse, Gunnar M, Van Geet, Chris, Freson, Kathleen

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Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years von Goemans, Nathalie, Klingels, Katrijn, van den Hauwe, Marleen, Boons, Stefanie, Verstraete, Liese, Peeters, Charlotte, Feys, Hilde, Buyse, Gunnar

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Dystrophin deficiency leads to dysfunctional glutamate clearance in iPSC derived astrocytes von Patel, Abdulsamie M., Wierda, Keimpe, Thorrez, Lieven, van Putten, Maaike, De Smedt, Jonathan, Ribeiro, Luis, Tricot, Tine, Gajjar, Madhavsai, Duelen, Robin, Van Damme, Philip, De Waele, Liesbeth, Goemans, Nathalie, Tanganyika-de Winter, Christa, Costamagna, Domiziana, Aartsma-Rus, Annemieke, van Duyvenvoorde, Hermine, Sampaolesi, Maurilio, Buyse, Gunnar M., Verfaillie, Catherine M.

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Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy von Meier, Thomas, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, Mayer, Oscar H, Buyse, Gunnar M

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Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy von Buyse, Gunnar M., Voit, Thomas, Schara, Ulrike, Straathof, Chiara S.M., D'Angelo, Maria Grazia, Bernert, Günther, Cuisset, Jean‐Marie, Finkel, Richard S., Goemans, Nathalie, Rummey, Christian, Leinonen, Mika, Mayer, Oscar H., Spagnolo, Paolo, Meier, Thomas, McDonald, Craig M.

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Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: Results from a 12 month, double-blind, randomized placebo-controlled trial von Buyse, Gunnar M, Goemans, Nathalie, van den Hauwe, Marleen, Thijs, Daisy, de Groot, Imelda J.M, Schara, Ulrike, Ceulemans, Berten, Meier, Thomas, Mertens, Luc

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Activin A Modulates CRIPTO-1/HNF4α+ Cells to Guide Cardiac Differentiation from Human Embryonic Stem Cells von Thorrez, Lieven, Sampaolesi, Maurilio, Buyse, Gunnar M., Sipido, Karin R., Roderick, Llewelyn, De Waele, Liesbeth, de Schaetzen, Nathalie, Patel, Abdulsamie, Gilbert, Guillaume, Duelen, Robin, Verfaillie, Catherine

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Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance von Buyse, Gunnar M., Van der Mieren, Gerry, Erb, Michael, D'hooge, Jan, Herijgers, Paul, Verbeken, Erik, Jara, Alejandro, Van Den Bergh, An, Mertens, Luc, Courdier-Fruh, Isabelle, Barzaghi, Patrizia, Meier, Thomas

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Regulated granule trafficking in platelets and neurons: A common molecular machinery von Goubau, Christophe, Buyse, Gunnar M, Di Michele, Michela, Van Geet, Chris, Freson, Kathleen

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Efficacy of Idebenone to Preserve Respiratory Function above Clinically Meaningful Thresholds for Forced Vital Capacity (FVC) in Patients with Duchenne Muscular Dystrophy von Mayer, Oscar H., Leinonen, Mika, Rummey, Christian, Meier, Thomas, Buyse, Gunnar M.

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