Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy von VANDENDRIESSCHE, T., THORREZ, L., ACOSTA‐SANCHEZ, A., PETRUS, I., WANG, L., MA, L., DE WAELE, L., IWASAKI, Y., GILLIJNS, V., WILSON, J. M., COLLEN, D., CHUAH, M. K. L.

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2024 update: European consensus statement on gene therapy for spinal muscular atrophy von Kirschner, Janbernd, Bernert, Günther, Butoianu, Nina, De Waele, Liesbeth, Fattal-Valevski, Aviva, Haberlova, Jana, Moreno, Teresa, Klein, Andrea, Kostera-Pruszczyk, Anna, Mercuri, Eugenio, Quijano-Roy, Susana, Sejersen, Thomas, Tizzano, Eduardo F., van der Pol, W Ludo, Wallace, Sean, Zafeiriou, Dimitrios, Ziegler, Andreas, Muntoni, Francesco, Servais, Laurent

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On the cutting edge: anterior transpetrosal approach – the middle fossa approach. Clinical application, surgical anatomy, and results von Mastronardi, L., De Waele, L., Fukushima, T.

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Myotonic dystrophy type 1: Long term natural history data von De Waele, L, Vanwesemael, C, Goemans, N

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Toward an appropriate neuropsychological assessment protocol for children with myotonic dystrophy type 1 von Geuens, S, Goemans, N, Thiery, E, Van Herck, J, Lemiere, J, De Waele, L

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The missing link von Maus, J, Naegels, S, Slabbynck, H, De Waele, L, Ruytjens, I

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Evaluation of three serum i-ELISAs using monoclonal antibodies and protein G as peroxidase conjugate for the diagnosis of bovine brucellosis von Saegerman, C, De Waele, L, Gilson, D, Godfroid, J, Thiange, P, Michel, P, Limbourg, B, Vo, T.K.-O, Limet, J, Letesson, J.-J, Berkvens, D

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P39 – 2771: Variable phenotype in epilepsy caused by KCNQ2 mutations von Jansen, K, Dereymaeker, A, De Waele, L, Lagae, L

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Baseline data from a European prospective and longitudinal natural history study of patients with type 2 and 3 spinal muscular atrophy – NatHis-SMA von Chabanon, A, Pereon, Y, Daron, A, Cances, C, Vuillerot, C, Fontaine, S, Goemans, N, De Waele, L, Laugel, V, Cuisset, J, Schara, U, Gangfuss, A, Gidaro, T, Gargaun, E, Marquet, A, Villeret, M, Phelep, A, Annoussamy, M, Servais, L

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P76 Bethlem myopathy presenting as a limb girdle muscular dystrophy von De Waele, L, Mayhew, A, Straub, V

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PP08.8 – 2833: Multiple sclerosis in Belgian children: A multicentric retrospective study von Verhelst, H, De Waele, L, Deconinck, N, Ceulemans, B, Willekens, B, Van Coster, R

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G.P.173 von De Waele, L, Braat, E, Vermeersch, P, Gheysens, O, Levtchenko, E, Pottel, H, Hoste, L, Goemans, N

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G.P.172 von De Waele, L, Braat, E, Vermeersch, P, Gheysens, O, Levtchenko, E, Pottel, H, Hoste, L, Goemans, N

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Treatment Patterns and Use of Resources in Patients With Tuberous Sclerosis Complex: Insights From the TOSCA Registry von Marques, Ruben, Belousoye, Elena, Benedik, Mirjana P, Carter, Tom, Cottin, Vincent, Curatolo, Paolo, Dahlin, Maria, D'Amato, Lisa, d'Augeres, Guillaume Beaure, de Vries, Petrus J, Ferreira, Jose C, Feucht, Martha, Fladrowski, Carla, Hertzberg, Christoph, Jozwiak, Sergiusz, Lawson, John A, Macaya, Alfons, Nabbout, Rima, O'Callaghan, Finbar, Qin, Jiong, Sander, Valentin, Sauter, Matthias, Shah, Seema, Takahashi, Yukitoshi, Touraine, Renaud, Youroukos, Sotiris, Zonnenberg, Bernard, Kingswood, John C, Jansen, Anna C, TOSCA Consortium and TOSCA Investigators

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Newly Diagnosed and Growing Subependymal Giant Cell Astrocytoma in Adults With Tuberous Sclerosis Complex: Results From the International TOSCA Study von Jansen, Anna C, Belousova, Elena, Benedik, Mirjana P, Carter, Tom, Cottin, Vincent, Curatolo, Paolo, D'Amato, Lisa, d'Augeres, Guillaume Beaure, de Vries, Petrus J, Ferreira, Jose C, Feucht, Martha, Fladrowski, Carla, Hertzberg, Christoph, Jozwiak, Sergiusz, Lawson, John A, Macaya, Alfons, Marques, Ruben, Nabbout, Rima, O'Callaghan, Finbar, Qin, Jiong, Sander, Valentin, Sauter, Matthias, Shah, Seema, Takahashi, Yukitoshi, Touraine, Renaud, Youroukos, Sotiris, Zonnenberg, Bernard, Kingswood, John C, TOSCA Consortium and TOSCA Investigators

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Clinical Characteristics of Subependymal Giant Cell Astrocytoma in Tuberous Sclerosis Complex von Jansen, Anna C, Belousova, Elena, Benedik, Mirjana P, Carter, Tom, Cottin, Vincent, Curatolo, Paolo, Dahlin, Maria, D'Amato, Lisa, d'Augeres, Guillaume Beaure, de Vries, Petrus J, Ferreira, Jose C, Feucht, Martha, Fladrowski, Carla, Hertzberg, Christoph, Jozwiak, Sergiusz, Lawson, John A, Macaya, Alfons, Marques, Ruben, Nabbout, Rima, O'Callaghan, Finbar, Qin, Jiong, Sander, Valentin, Sauter, Matthias, Shah, Seema, Takahashi, Yukitoshi, Touraine, Renaud, Youroukos, Sotiris, Zonnenberg, Bernard, Kingswood, John C, TOSCA Consortium

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P248 DuMAND checklist: a screening tool for behavioural difficulties in Duchenne muscular dystrophy von Geuens, S., Schroven, S., Lemiere, J., De Waele, L.

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PP15.1 – 2964: Long-term renal outcome of a large cohort of patients with tuberous sclerosis complex von De Waele, L, Van Hoeve, K, Janssens, P, Claes, K, Lagae, L, Levtchenko, E, Bammens, B, Mekahli, D, Jansen, A

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311P Development and pilot validation of the DuMAND checklist to screen for Duchenne muscular dystrophy-associated neurobehavioral difficulties (DuMAND) von Geuens, S., Goemans, N., Lemiere, J., Doorenweerd, N., De Waele, L.

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51Cr-EDTA plasma clearance in children: One, two, or multiple samples? von Pottel, Hans, Goffin, Karolien, De Waele, Liesbeth, Levtchenko, Elena, Delanaye, Pierre

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