Duchenne muscular dystrophy von Duan, Dongsheng, Goemans, Nathalie, Takeda, Shin’ichi, Mercuri, Eugenio, Aartsma-Rus, Annemieke

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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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Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy von Mendell, Jerry R., Goemans, Nathalie, Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Shao, James, Kaye, Edward M., Mercuri, Eugenio

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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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Neuromuscular Disorders in Children: A Multidisciplinary Approach to Management von Deconinck, Nicolas

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Gait classification for growing children with Duchenne muscular dystrophy von Vandekerckhove, Ines, Papageorgiou, Eirini, Hanssen, Britta, De Beukelaer, Nathalie, Van den Hauwe, Marleen, Goemans, Nathalie, Van Campenhout, Anja, De Waele, Liesbeth, De Groote, Friedl, Desloovere, Kaat

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Gene therapy for spinal muscular atrophy: hope and caution von Goemans, Nathalie

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Novel defects in collagen XII and VI expand the mixed myopathy/Ehlers–Danlos syndrome spectrum and lead to variant-specific alterations in the extracellular matrix von Delbaere, Sarah, Dhooge, Tibbe, Syx, Delfien, Petit, Florence, Goemans, Nathalie, Destrée, Anne, Vanakker, Olivier, De Rycke, Riet, Symoens, Sofie, Malfait, Fransiska

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A new strength assessment to evaluate the association between muscle weakness and gait pathology in children with cerebral palsy von Goudriaan, Marije, Nieuwenhuys, Angela, Schless, Simon-Henri, Goemans, Nathalie, Molenaers, Guy, Desloovere, Kaat

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Prognostic factors for changes in the timed 4-stair climb in patients with Duchenne muscular dystrophy, and implications for measuring drug efficacy: A multi-institutional collabor... von Goemans, Nathalie, Wong, Brenda, Van den Hauwe, Marleen, Signorovitch, James, Sajeev, Gautam, Cox, David, Landry, John, Jenkins, Madeline, Dieye, Ibrahima, Yao, Zhiwen, Hossain, Intekhab, Ward, Susan J

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051 von van Deutekom, Judith C, Janson, Anneke A, Ginjaar, Ieke B, Frankhuizen, Wendy S, Aartsma-Rus, Annemieke, Bremmer-Bout, Mattie, den Dunnen, Johan T, Koop, Klaas, van der Kooi, Anneke J, Goemans, Nathalie M, de Kimpe, Sjef J, Ekhart, Peter F, Venneker, Edna H, Platenburg, Gerard J, Verschuuren, Jan J, van Ommen, Gert-Jan B

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Muscle weakness has a limited effect on motor control of gait in Duchenne muscular dystrophy von Vandekerckhove, Ines, De Beukelaer, Nathalie, Van den Hauwe, Marleen, Shuman, Benjamin R, Steele, Katherine M, Van Campenhout, Anja, Goemans, Nathalie, Desloovere, Kaat, Goudriaan, Marije

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Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials von McDonald, Craig M, Signorovitch, James, Mercuri, Eugenio, Niks, Erik H, Wong, Brenda, Fillbrunn, Mirko, Sajeev, Gautam, Yim, Erica, Dieye, Ibrahima, Miller, Debra, Ward, Susan J, Goemans, Nathalie

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Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53 von Brogna, Claudia, Coratti, Giorgia, Pane, Marika, Ricotti, Valeria, Messina, Sonia, D'Amico, Adele, Bruno, Claudio, Vita, Gianluca, Berardinelli, Angela, Mazzone, Elena, Magri, Francesca, Ricci, Federica, Mongini, Tiziana, Battini, Roberta, Bello, Luca, Pegoraro, Elena, Baranello, Giovanni, Previtali, Stefano C, Politano, Luisa, Comi, Giacomo P, Sansone, Valeria A, Donati, Alice, Bertini, Enrico, Muntoni, Francesco, Goemans, Nathalie, Mercuri, Eugenio

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Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy von Goemans, Nathalie, Vanden Hauwe, Marleen, Signorovitch, James, Swallow, Elyse, Song, Jinlin

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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy von Straub, Volker, MD, Balabanov, Pavel, PhD, Bushby, Kate, MD, Ensini, Monica, PhD, Goemans, Nathalie, MD, De Luca, Annamaria, PhD, Pereda, Alejandra, Hemmings, Robert, Campion, Giles, MD, Kaye, Edward, MD, Arechavala-Gomeza, Virginia, PhD, Goyenvalle, Aurelie, PhD, Niks, Erik, MD, Veldhuizen, Olav, Furlong, Pat, Stoyanova-Beninska, Violeta, MD, Wood, Matthew J, PhD, Johnson, Alex, Mercuri, Eugenio, MD, Muntoni, Francesco, MD, Sepodes, Bruno, PhD, Haas, Manuel, PharmD, Vroom, Elizabeth, DMD, Aartsma-Rus, Annemieke, Dr

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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study von Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Done, Nicolae, Yao, Zhiwen, Goemans, Nathalie, McDonald, Craig, Mercuri, Eugenio, Niks, Erik H, Wong, Brenda, Vandenborne, Krista, Straub, Volker, de Groot, Imelda J M, Tian, Cuixia, Manzur, Adnan, Dieye, Ibrahima, Lane, Henry, Ward, Susan J, Servais, Laurent

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Illness Perceptions and Illness Identity in Adolescents and Emerging Adults With Neuromuscular Disorders von Geuens, Sam, Leyen, Kathelijne, Raymaekers, Koen, Prikken, Sofie, Willen, Joanna, Goemans, Nathalie, De Waele, Liesbeth, Lemiere, Jurgen, Luyckx, Koen

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How glucocorticoids change life in Duchenne muscular dystrophy von Goemans, Nathalie

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