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

por Goemans, Nathalie, vanden Hauwe, Marleen, Signorovitch, James, Swallow, Elyse, Song, Jinlin
Publicado 2016

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

por Goudriaan, Marije, Nieuwenhuys, Angela, Schless, Simon-Henri, Goemans, Nathalie, Molenaers, Guy, Desloovere, Kaat
Publicado 2018

Six-Minute Walk Test: Reference Values and Prediction Equation in Healthy Boys Aged 5 to12 Years

por Goemans, Nathalie, Klingels, Katrijn, van den Hauwe, Marleen, Boons, Stefanie, Verstraete, Liese, Peeters, Charlotte, Feys, Hilde, Buyse, Gunnar
Publicado 2013

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

por Goudriaan, Marije, Shuman, Benjamin R., Steele, Katherine M., Van den Hauwe, Marleen, Goemans, Nathalie, Molenaers, Guy, Desloovere, Kaat
Publicado 2018

International workshop on assessment of upper limb function in Duchenne Muscular Dystrophy: Rome, 15–16 February 2012

por Mercuri, Eugenio, McDonald, Craig, Mayhew, Anna, Florence, Julaine, Mazzone, Elena, Bianco, Flaviana, Decostre, Valerie, Servais, Laurent, Ricotti, Valeria, Goemans, Nathalie, Vroom, Elizabeth
Publicado 2012

Helical mutations in type I collagen that affect the processing of the amino-propeptide result in an Osteogenesis Imperfecta/Ehlers-Danlos Syndrome overlap syndrome

por Malfait, Fransiska, Symoens, Sofie, Goemans, Nathalie, Gyftodimou, Yolanda, Holmberg, Eva, López-González, Vanesa, Mortier, Geert, Nampoothiri, Sheela, Petersen, Michael Bjorn, De Paepe, Anne
Publicado 2013

Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls

por Mendell, Jerry R., Khan, Navid, Sha, Nanshi, Eliopoulos, Helen, McDonald, Craig M., Goemans, Nathalie, Mercuri, Eugenio, Lowes, Linda P., Alfano, Lindsay N.
Publicado 2021

Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients

por Lochmüller, Hanns, Evans, David, Farwell, Wildon, Finkel, Richard, Goemans, Nathalie, de Lemus, Mencia, Matyushenko, Vitaliy, Muntoni, Francesco, Ouillade, Marie-Christine, Schwersenz, Inge, Wilson, Petra
Publicado 2018

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

por Vandekerckhove, Ines, De Beukelaer, Nathalie, Van den Hauwe, Marleen, Shuman, Benjamin R., Steele, Katherine M., Van Campenhout, Anja, Goemans, Nathalie, Desloovere, Kaat, Goudriaan, Marije
Publicado 2020

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

por Goemans, Nathalie, Signorovitch, James, Sajeev, Gautam, Yao, Zhiwen, Gordish-Dressman, Heather, McDonald, Craig M., Vandenborne, Krista, Miller, Debra, Ward, Susan J., Mercuri, Eugenio
Publicado 2020

Human iPSC model reveals a central role for NOX4 and oxidative stress in Duchenne cardiomyopathy

por Duelen, Robin, Costamagna, Domiziana, Gilbert, Guillaume, Waele, Liesbeth De, Goemans, Nathalie, Desloovere, Kaat, Verfaillie, Catherine M., Sipido, Karin R., Buyse, Gunnar M., Sampaolesi, Maurilio
Publicado 2022

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era

por Walter, Maggie C., Chiriboga, Claudia, Duong, Tina, Goemans, Nathalie, Mayhew, Anna, Ouillade, Laëtitia, Oskoui, Maryam, Quinlivan, Ros, Vázquez-Costa, Juan F., Vissing, John, Servais, Laurent
Publicado 2021

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...

por 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.
Publicado 2020

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

por 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
Publicado 2016

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

por 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
Publicado 2022

Instrumented strength assessment in typically developing children and children with a neural or neuromuscular disorder: A reliability, validity and responsiveness study

por Verreydt, Ineke, Vandekerckhove, Ines, Stoop, Elze, Peeters, Nicky, van Tittelboom, Vanessa, Van de Walle, Patricia, Van den Hauwe, Marleen, Goemans, Nathalie, De Waele, Liesbeth, Van Campenhout, Anja, Hanssen, Britta, Desloovere, Kaat
Publicado 2022

European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

por Kirschner, Janbernd, Butoianu, Nina, Goemans, Nathalie, Haberlova, Jana, Kostera-Pruszczyk, Anna, Mercuri, Eugenio, van der Pol, W. Ludo, Quijano-Roy, Susana, Sejersen, Thomas, Tizzano, Eduardo F., Ziegler, Andreas, Servais, Laurent, Muntoni, Francesco
Publicado 2020

Functional and Clinical Outcomes Associated with Steroid Treatment among Non-ambulatory Patients with Duchenne Muscular Dystrophy(1)

por McDonald, Craig M., Mayer, Oscar H., Hor, Kan N., Miller, Debra, Goemans, Nathalie, Henricson, Erik K., Marden, Jessica R., Freimark, Jonathan, Lane, Henry, Zhang, Adina, Frean, Molly, Trifillis, Panayiota, Koladicz, Karyn, Signorovitch, James
Publicado 2023

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

por Victor, Ronald G., Sweeney, H. Lee, Finkel, Richard, McDonald, Craig M., Byrne, Barry, Eagle, Michelle, Goemans, Nathalie, Vandenborne, Krista, Dubrovsky, Alberto L., Topaloglu, Haluk, Miceli, M. Carrie, Furlong, Pat, Landry, John, Elashoff, Robert, Cox, David
Publicado 2017

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

por McDonald, Craig M., Shieh, Perry B., Abdel-Hamid, Hoda Z., Connolly, Anne M., Ciafaloni, Emma, Wagner, Kathryn R., Goemans, Nathalie, Mercuri, Eugenio, Khan, Navid, Koenig, Erica, Malhotra, Jyoti, Zhang, Wenfei, Han, Baoguang, Mendell, Jerry R.
Publicado 2021