Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives von Dangouloff, Tamara, Servais, Laurent

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P73 Newborn screening programs for spinal muscular atrophy worldwide: are we there yet? von Vrščaj, E., Dangouloff, T., Osredkar, D., Servais, L.

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SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES von Dangouloff, T., Servais, L., Hiligsmann, M.

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SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES von Dangouloff, T., Servais, L., Hiligsmann, M.

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SMA CLINICAL DATA von Dangouloff, T., Vrscaj, E., Servais, L., Osredkar, D.

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SMA CLINICAL DATA: EP.254 Newborn screening for spinal muscular atrophy in the world today von Dangouloff, T., Vrscaj, E., Servais, L., Osredkar, D.

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O10 Universal genomic newborn screening for early, treatable, and severe conditions- including 33 genes of NMD: Baby Detect von Dangouloff, T., Hovhannesyan, K., Piazzon, F., Mashhadizadeh, D., Helou, L., Palmeira, L., Boemer, F., Servais, L.

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SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES: P.191 Preliminary data for the cost-effectiveness assessment of the newborn screening for SMA in Belgium von Dangouloff, T., Servais, L., Hiligsmann, M.

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SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES: P.187 Systematic literature review of the economic burden and economic evaluations in spinal muscular atrophy von Dangouloff, T., Servais, L., Hiligsmann, M.

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P.44 Cost-effectiveness of spinal muscular atrophy newborn screening in Belgium von Dangouloff, T., Thokala, P., Daron, A., Delstanche, S., Servais, L., Hiligsmann, M.

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Cerebral blood flow and acute episodes of Leigh syndrome in neurometabolic disorders von Loiselet, Klervie, Ruzzenente, Benedetta, Roux, Charles‐Joris, Barcia, Giulia, Pennisi, Alessandra, Desguerre, Isabelle, Rötig, Agnès, Munnich, Arnold, Boddaert, Nathalie, Levy, Raphaël, Dangouloff‐Ros, Volodia, Grévent, David, De Lonlay, Pascale

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145P Efficacy of SMA NBS: 4-year comparative study with control group von Dangouloff, T., De Waele, L., Beysen, D., Smeets, N., Vanlander, A., Benmhammed, N., Tychon, C., Daron, A., Deconinck, N., Dontaine, P., Servais, L.

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SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES von Dangouloff, T., Hiligsmann, M., Caberg, J., Boemer, F., Servais, L.

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SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES: P.97Development of a decision-analytic model for the economic evaluation of newborn screening for spinal muscular atrophy von Dangouloff, T., Hiligsmann, M., Caberg, J., Boemer, F., Servais, L.

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244th ENMC international workshop: Newborn screening in spinal muscular atrophy May 10–12, 2019, Hoofdorp, The Netherlands von Dangouloff, Tamara, Burghes, Arthur, Tizzano, Eduardo F., Servais, Laurent, Dangouloff, Tamara, Burghes, Arthur, Bertini, Enrico, Boemer, François, Hiligsmann, Mickaël, Mueller-Felber, Wolfgang, Tiziano, Danilo, Young, Philip, Tizzano, Eduardo F., Servais, Laurent, Germanenko, Olga, de Lemus, Mencia, Ouillade, Laetitia, Rucinski, Kasper, Stephenson, Kristin, Farwell, Wildon, Gorni, Ksenija, Hoffmann, F., Hjort, Mikael, Kausar, Imran

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ERN GENTURIS guidelines on constitutional mismatch repair deficiency diagnosis, genetic counselling, surveillance, quality of life, and clinical management von Colas, Chrystelle, Guerrini-Rousseau, Léa, Suerink, Manon, Gallon, Richard, Kratz, Christian P., Ayuso, Éloïse, Brugières, Laurence, Wimmer, Katharina

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P.225Reducing the diagnosis time of neonatal screening by optimizing the screening process: the southern Belgian experience von Dangouloff, T., Boemer, F., Caberg, J., Di Fiore, S., Beckers, P., Marie, S., Marcelis, L., Servais, L.

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Neuroimaging manifestations in children with SARS-CoV-2 infection: a multinational, multicentre collaborative study von Lindan, Camilla E, Mankad, Kshitij, Ram, Dipak, Kociolek, Larry K, Silvera, V Michelle, Boddaert, Nathalie, Stivaros, Stavros Michael, Palasis, Susan, Akhtar, Sameen, Alden, Douglas, Amonkar, Suraj, Aouad, Pascale, Aubart, Mélodie, Bacalla, Jose Alejandro, Barbosa, Alcino A, Basmaci, Romain, Berteloot, Laureline, Blauwblomme, Thomas, Brun, Gilles, Carney, Olivia, Chareyre, Judith, Chéron, Gérard, Coimbra, Pablo Picasso De Araujo, Dangouloff-Ros, Volodia, D'Arco, Felice, Dineen, Rob, De-Pontual, Loic, Desguerre, Isabelle, Elfallal, Wissam, Evans, D. Gareth, Ferraciolli, Suely Fazio, Girard, Nadine, Gonçalves, Fabrício Guimarães, Gonzalez, Ivan, Grant, P. Ellen, Grévent, David, Guimaraes, Carolina Valduga de Alencastro, Hassell, Jane, Hirata, Fabiana C.C., Kamaly-Asl, Ian, Jacob, Jeffrey, Jackson, Kandise, Jones, Blaise V., Joseph, Robin, Jung, Ah Young, Kashgari, Amna, Kilday, John-Paul, Kirsch, Alyssa, Kossorotoff, Manoelle, Krishnan, Anant, Kulkarni, Shilpa, Leruez-Vill, Marianne, Lesage, Fabrice, Levy, Raphaël, Li, Yi, Lima, Carol Cavalcante de Vasconcelos, Lingappa, Lokesh, Löbel, Ulrike, Lopez-Alberola, Roberto, Lucato, Leandro Tavares, Moreira, Daniela Duarte, Murnick, Jonathan G., Nahmani, Sarah, Pagariya, Shubra, Pavaine, Julija, Philbrook, Bryan, Piovesan, Ana Cláudia, Poisson, Kelsey E., Reddy, Nihaal, Riley, Phil, Romsauerova, Andrea, Roux, Charlies-Joris, Rugilo, Carlos, Saigal, Gaurav, Salvador, Gabriel Lucca de Oliveira, Seidenwurm, David, Sermet-Gaudelus, Isabelle, Sidpra, Jai, Sudhakar, Sniya Valsa, Toronchik, María Sol, Vézina, Gilbert

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European recommendations on practices in pediatric neuroradiology: consensus document from the European Society of Neuroradiology (ESNR), European Society of Paediatric Radiology (... von Rossi, Andrea, Argyropoulou, Maria, Zlatareva, Dora, Boulouis, Gregoire, Pizzini, Francesca B., van den Hauwe, Luc, Raissaki, Maria, Pruvo, Jean-Pierre, Rosendahl, Karen, Hoffmann, Chen, Sundgren, Pia C.

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Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium von Dangouloff, T, Thokala, P, Stevenson, M.D, Deconinck, N, D'Amico, A, Daron, A, Delstanche, S, Servais, L, Hiligsmann, M

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