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Duchenne muscular dystrophy
Author / Creator:Duan, Dongsheng ; Goemans, Nathalie ; Takeda, Shin’ichi ; Mercuri, Eugenio ; Aartsma-Rus, Annemieke
Publisher:London: Nature Publishing Group UK
Journal title:Nature reviews. Disease primers, 2021-02, Vol.7 (1), p.13, Article 13
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Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study
Author / Creator: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
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-09, Vol.11 (9), p.e0161955-e0161955
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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
Author / Creator: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
Publisher:Waltham, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2011-04, Vol.364 (16), p.1513-1522
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A new strength assessment to evaluate the association between muscle weakness and gait pathology in children with cerebral palsy
Author / Creator:Goudriaan, Marije ; Nieuwenhuys, Angela ; Schless, Simon-Henri ; Goemans, Nathalie ; Molenaers, Guy ; Desloovere, Kaat
Publisher:United States: Public Library of Science
Journal title:PloS one, 2018-01, Vol.13 (1), p.e0191097-e0191097
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Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy
Author / Creator:Vandekerckhove, Ines ; Van den Hauwe, Marleen ; Dewit, Tijl ; Molenberghs, Geert ; Goemans, Nathalie ; De Waele, Liesbeth ; Van Campenhout, Anja ; De Groote, Friedl ; Desloovere, Kaat
Publisher:United States: Public Library of Science
Journal title:PloS one, 2025-03, Vol.20 (3), p.e0307007
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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
Author / Creator:Delbaere, Sarah ; Dhooge, Tibbe ; Syx, Delfien ; Petit, Florence ; Goemans, Nathalie ; Destrée, Anne ; Vanakker, Olivier ; De Rycke, Riet ; Symoens, Sofie ; Malfait, Fransiska
Publisher:New York: Nature Publishing Group US
Journal title:Genetics in medicine, 2020-01, Vol.22 (1), p.112-123
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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 collaboration
Author / Creator: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.
Publisher:San Francisco: Public Library of Science
Journal title:PloS one, 2020-06, Vol.15 (6), p.e0232870-e0232870
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Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy
Author / Creator:Goemans, Nathalie ; vanden Hauwe, Marleen ; Signorovitch, James ; Swallow, Elyse ; Song, Jinlin
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-10, Vol.11 (10), p.e0164684
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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
Author / Creator: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
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2007-12, Vol.357 (26), p.2677-2686
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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 trial
Author / Creator: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
Publisher:England: Elsevier Ltd
Journal title:The Lancet (British edition), 2015-05, Vol.385 (9979), p.1748-1757
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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
Author / Creator: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
Publisher:United States: Public Library of Science
Journal title:PloS one, 2019-06, Vol.14 (6), p.e0218683
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Gait classification for growing children with Duchenne muscular dystrophy
Author / Creator: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
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2024-05, Vol.14 (1), p.10828-10828, Article 10828
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Muscle weakness but also contractures contribute to the progressive gait pathology in children with Duchenne muscular dystrophy: a simulation study
Author / Creator:Vandekerckhove, Ines ; D’Hondt, Lars ; Gupta, Dhruv ; Van Den Bosch, Bram ; Van den Hauwe, Marleen ; Goemans, Nathalie ; De Waele, Liesbeth ; Van Campenhout, Anja ; Desloovere, Kaat ; De Groote, Friedl
Publisher:England: BioMed Central Ltd
Journal title:Journal of neuroengineering and rehabilitation, 2025-05, Vol.22 (1), p.103-20, Article 103
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Muscle weakness has a limited effect on motor control of gait in Duchenne muscular dystrophy
Author / Creator:Vandekerckhove, Ines ; De Beukelaer, Nathalie ; Van den Hauwe, Marleen ; Shuman, Benjamin R. ; Steele, Katherine M. ; Van Campenhout, Anja ; Goemans, Nathalie ; Desloovere, Kaat ; Goudriaan, Marije
Publisher:United States: Public Library of Science
Journal title:PloS one, 2020-09, Vol.15 (9), p.e0238445
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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy
Author / Creator: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
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2016-07, Vol.15 (8), p.882-890
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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint
Author / Creator:Servais, Laurent ; Strijbos, Paul ; Poleur, Margaux ; Mirea, Andrada ; Butoianu, Nina ; Sansone, Valeria A. ; Vuillerot, Carole ; Schara-Schmidt, Ulrike ; Scoto, Mariacristina ; Seferian, Andreea M. ; Previtali, Stefano C. ; Tulinius, Már ; Nascimento, Andrés ; Furlong, Pat ; Singh, Teji ; Dreghici, Roxana Donisa ; Goemans, Nathalie ; Mercuri, Eugenio ; Straub, Volker ; Ormazabal, Maitea Guridi ; Braid, Jessica ; Muntoni, Francesco ; Tricot, Alexis ; Annoussamy, Mélanie ; Eggenspieler, Damien
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2024-11, Vol.14 (1), p.29681-12, Article 29681
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Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years
Author / Creator:Goemans, Nathalie ; Klingels, Katrijn ; van den Hauwe, Marleen ; Boons, Stefanie ; Verstraete, Liese ; Peeters, Charlotte ; Feys, Hilde ; Buyse, Gunnar
Publisher:United States: Public Library of Science (PLoS)
Journal title:PloS one, 2013, Vol.8 (12), p.e84120-e84120
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North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up
Author / Creator:Coratti, Giorgia ; Pane, Marika ; Brogna, Claudia ; 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 ; Hogrel, Jean Yves ; Straub, Volker ; De Lucia, Silvana ; Niks, Erik ; Servais, Laurent ; De Groot, Imelda ; Chesshyre, Mary ; Bertini, Enrico ; Goemans, Nathalie ; Muntoni, Francesco ; Mercuri, Eugenio
Publisher:San Francisco: Public Library of Science
Journal title:PloS one, 2021-06, Vol.16 (6), p.e0253882-e0253882
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Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy
Author / Creator:Vandekerckhove, Ines ; Van den Hauwe, Marleen ; De Beukelaer, Nathalie ; Stoop, Elze ; Goudriaan, Marije ; Delporte, Margaux ; Molenberghs, Geert ; Van Campenhout, Anja ; De Waele, Liesbeth ; Goemans, Nathalie ; De Groote, Friedl ; Desloovere, Kaat
Publisher:Lausanne: Frontiers Research Foundation
Journal title:Frontiers in human neuroscience, 2022-06
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Non-neural Muscle Weakness Has Limited Influence on Complexity of Motor Control during Gait
Author / Creator:Goudriaan, Marije ; Shuman, Benjamin R. ; Steele, Katherine M. ; Van den Hauwe, Marleen ; Goemans, Nathalie ; Molenaers, Guy ; Desloovere, Kaat
Publisher:Switzerland: Frontiers Research Foundation
Journal title:Frontiers in human neuroscience, 2018-01, Vol.12, p.5-5
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Showing 1 to 20 of 57 results

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Duchenne muscular dystrophy

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Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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A new strength assessment to evaluate the association between muscle weakness and gait pathology in children with cerebral palsy

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Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy

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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

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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 collaboration

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Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

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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 trial

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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

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Gait classification for growing children with Duchenne muscular dystrophy

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Muscle weakness but also contractures contribute to the progressive gait pathology in children with Duchenne muscular dystrophy: a simulation study

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Muscle weakness has a limited effect on motor control of gait in Duchenne muscular dystrophy

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Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint

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Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years

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North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

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Longitudinal Alterations in Gait Features in Growing Children With Duchenne Muscular Dystrophy

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Non-neural Muscle Weakness Has Limited Influence on Complexity of Motor Control during Gait

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