280P Technology-enabled assessment of cognition to facilitate democratization of brain health in Duchenne muscular dystrophy von Thangarajh, M.

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Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy von Morales, Juan Francisco, Corey, Diane, Larkindale, Jane, Romero, Klaus, Schmidt, Stephan, Cregan, M, Johnson, L, Han, J, Joyce, N, Nicorici, A, Reddy, D, Mah, J, Haig, T, Sanchez, K, Alhander, A, Ekstrom, A, Gustafsson, A, Kroksmark, A, Wahlgren, L, Brody, N, Drogo, B, Leach, M, Tesi‐Rocha, C, Birkmeier, M, Toles, A, Carroll, K, DeValle, K, Kennedy, R, Rodriguez, V, Nevo, Y, Adani, R, BarLeve, A, Chen‐Joseph, L, Daana, M, Panteleyev‐Yitshak, V, Yaffe, D, Levi, L, Bendixen, R, Karnavas, K, Matthews, C, Niizawa, G, Weimer, J, Florence, J, Gadeken, R, Golumbak, P, Malkus, B, Pestronk, A, Schierbecker, J, Seiner, C, Wulf, C, Blair, S, Grillo, B, Bertorini, T, Barrett‐Adair, M, Benzel, C, Clift, J, Holloway, J, Igarashi, M, Kiphut, F, Parker, A, Phillips, A, Young, R, North, K, Gabriel, N, Harman, M, Rose, K, Kolski, H, Chen, L, Beneggi, M, Capone, L, Molteni, A, Morettini, V, Gupta, A, Knight, A, Lott, B, McNeil, R, Orozco, G, Chambers, G, Day, J, Dalton, J, Erickson, A, Marsh, J, Naughton, C, Hoffman, A, Korn‐Petersen, W, Deliz, B, Fuste, P, Luciano, C, Torres, J, Ahmed, M, Arrieta, A, Brown‐Caines, T, Carty, C, Duong, T, Feng, J, Hu, F, Hunegs, L, Zimmerman, A, Nuckolls, Glen, Carifi, Emily

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286P Analysis of fluid cognition in boys with Duchenne muscular dystrophy von Arun, S., Kaat, A., Thangarajh, M.

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Proliferation-inducing Ligand (APRIL) is Expressed by Astrocytes and is Increased in Multiple Sclerosis von Thangarajh, M, Masterman, T, Hillert, J, Moerk, S, Jonsson, R

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Lipid-specific immunoglobulin M in CSF predicts adverse long-term outcome in multiple sclerosis von Thangarajh, M, Gomez-Rial, J, Hedström, AK, Hillert, J, Alvarez-Cermeño, JC, Masterman, T, Villar, LM

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249th ENMC International Workshop: The role of brain dystrophin in muscular dystrophy: Implications for clinical care and translational research, Hoofddorp, The Netherlands, Novemb... von Hendriksen, Jos G.M., Thangarajh, Mathula, Kan, Hermien E., Muntoni, Francesco, Aoki, Dr. Y., Collin, Dr P., Colvin, Dr M., Doorenweerd, Dr N., Ferlini, Prof A., Goyenvalle, Dr A., Hendriksen, Dr J.J., Hoskin, Dr J., Kan, Dr H.E., Lamy, Mr F., Maresh, Dr K., Muntoni, Prof F., Niks, Dr E.H., Schara, Prof U., Skuse, Prof D., Straub, Prof V., Takeda, Prof S., Thangarajh, Dr M., Truba, Dr N., Tyagarajan, Prof S., Vaillend, Dr C., van Putten, Dr M., Vissing, Prof J., Vroom, E.

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Cytoplasmic body pathology in severe ACTA1 -myopathy in the absence of typical nemaline-rod histology von Donkervoort, S, Chan, S, Bradley, N, Foley, A, Nguyen, D, Hu, Y, Leach, M, Thangarajh, M, Reyes, C, Nance, J, Moore, S, Bönnemann, C

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366P Social Vulnerability in Duchenne Muscular Dystrophy: The Impact on Boys and Carrier Mothers von Penumalee, V., Karra, H., Javalkar, S., Arun, S., Kaat, A., Thangarajh, M.

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378P Geospatial driver of cognition in biological mothers of sons with dystrophinopathy von Javalkar, S., Penumalee, V., Arun, S., Karra, H., Kaat, A., Thangarajh, M.

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DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study von Bello, Luca, Morgenroth, Lauren P, Gordish-Dressman, Heather, Hoffman, Eric P, McDonald, Craig M, Cirak, Sebahattin

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Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study von McDonald, Craig M, Henricson, Erik K, Abresch, Richard T, Duong, Tina, Joyce, Nanette C, Hu, Fengming, Clemens, Paula R, Hoffman, Eric P, Cnaan, Avital, Gordish-Dressman, Heather, Vishwanathan, Vijay, Chidambaranathan, S, Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Tulinius, Mar, Cnaan, Avital, Morgenroth, Lauren P., Leshner, Robert, Tesi-Rocha, Carolina, Thangarajh, Mathula, Duong, Tina, Kornberg, Andrew, Ryan, Monique, Nevo, Yoram, Dubrovsky, Alberto, Clemens, Paula R., Abdel-Hamid, Hoda, Connolly, Anne M., Pestronk, Alan, Teasley, Jean, Bertorin, Tulio E., Webster, Richard, Kolski, Hanna, Kuntz, Nancy, Driscoll, Sherilyn, Bodensteiner, John B., Carlo, Jose, Gorni, Ksenija, Lotze, Timothy, Day, John W., Karachunski, Peter, Henricson, Erik K., Abresch, Richard T., Joyce, Nanette C., McDonald, Craig M.

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expression of BAFF-binding receptors is not altered in multiple sclerosis or myasthenia gravis von Thangarajh, M, Kisiswa, L, Pirskanen, R, Hillert, J

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P.279Pragmatic cognitive and psychosocial health monitoring in clinical practice in Duchenne muscular dystrophy von Thangarajh, M., Kaat, A., Hardy, K., Wagner, K.

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Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids von McDonald, Craig M., Gordish-Dressman, Heather, Henricson, Erik K., Duong, Tina, Joyce, Nanette C., Jhawar, Sanjay, Leinonen, Mika, Hsu, Fengming, Connolly, Anne M., Cnaan, Avital, Abresch, Richard T., Dubrovsky, A., Kornberg, A., Ryan, M., Webster, R., Biggar, W.D., McAdam, L.C., Mah, J.K., Kolski, H., Vishwanathan, V., Chidambaranathan, S., Nevo, Y., Gorni, K., Carlo, J., Tulinius, M., Lotze, T., Bertorini, T.E., Day, J.W., Karachunski, P., Clemens, P.R., Abdel-Hamid, H., Teasley, J., Kuntz, N., Driscoll, S., Bodensteiner, J.B., Connolly, A.M., Pestronk, A., Abresch, R.T., Henricson, E.K., Joyce, N.C., McDonald, C.M., Cnaan, A., Morgenroth, L.P., Leshner, R., Tesi-Rocha, C., Thangarajh, M., Duong, T.

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P.14 Analysis of the longitudinal CINRG Becker natural history study dataset von Clemens, P., Gordish-Dressman, H., Niizawa, G., Gorni, K., Guglieri, M., Connolly, A., Wicklund, M., Bertorini, T., Mah, J., Thangarajh, M., Smith, E., Kuntz, N., McDonald, C., Henricson, E., Upadhyayula, S., Byrne, B., Manousakis, G., Harper, A., Iannaccone, S., Dang, U.

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Low-grade gliomas as neurodevelopmental disorders: insights from mouse models of neurofibromatosis-1 von Thangarajh, M., Gutmann, D. H.

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Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study von Mercuri, Eugenio, Muntoni, Francesco, Osorio, Andres Nascimento, Tulinius, Mar, Buccella, Filippo, Morgenroth, Lauren P., Gordish-Dressman, Heather, Jiang, Joel, Trifillis, Panayiota, Zhu, Lin, Kristensen, Allan, Santos, Claudio L., Henricson, Erik K., McDonald, Craig M., Desguerre, Isabelle, Bernert, G., Gosk-Tomek, M., Ille, A., Kellersmann, A., Weiss, S., Pilshofer, Balintova, Z., Danhofer, P., Fabulova, P., Jurikova, L., Fuchsova, P., Haberlova, J., Laffargue, F., Sarret, C., Pontier, B., Bellance, R., Sarrazin, E., Sabouraud, P., Magot, A., Mercier, S., Pereon, Y., Cuisset, J. M., Coopman-Degryse, S., Enaud, E., Jacquemont, M. L., Perville, A., Renouil, M., Trommsdorff, Verheulpen, D., Fontaine-Carbonnel, S., Vuillerot, C., Peudenier, S., Ropars, J., Audic, F., Chabrol, B., Chabrier, S., Gousse, G., Lagrue, E., Aragon, K., Barnerias, C., Brande, L., De Lucia, S., Desguerre, Gidaro, T., Seferian, A., Servais, L., Laugel, Espil-Taris, C., Mecili, H., Raffo, E., Ragot-Mandry, S., Borrell, S., Kirschner, J., Gangfuss, A., Henrich, M., Koelbel, H., Schara, U., Spoenemann, N., Temme, E., Seeger, J., Hirsch, A., Denecke, J., Johannsen, J., Neu, A., Osinski, D., Ruegner, S., Schuessler, S., Trollmann, R., Kaindl, A., Schneider, J. B., Stoltenburg, C., Weiss, C., Schreiber, G., Hahn, A., Grzybowski (Giessen), M., Pavlidou, E., Pavlou, E., Dobner, S., Liptai, Z., Dor, T., Brogna, C., Catteruccia, M., D'Amico, A., Mercuri, E., Pane, M.

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Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy von Heier, Christopher R., Zhang, Aiping, Nguyen, Nhu Y., Tully, Christopher B., Panigrahi, Aswini, Gordish-Dressman, Heather, Pandey, Sachchida Nand, Guglieri, Michela, Ryan, Monique M., Clemens, Paula R., Thangarajh, Mathula, Webster, Richard, Smith, Edward C., Connolly, Anne M., McDonald, Craig M., Karachunski, Peter, Tulinius, Mar, Harper, Amy, Mah, Jean K., Fiorillo, Alyson A., Chen, Yi-Wen

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Increased B Cell Survival and Preferential Activation of the Memory Compartment by a Malaria Polyclonal B Cell Activator von Donati, Daria, Mok, Bobo, Chene, Arnaud, Xu, Hong, Thangarajh, Mathula, Glas, Rickard, Chen, Qijun, Wahlgren, Mats, Bejarano, Maria Teresa

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Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study von Bello, Luca, Gordish-Dressman, Heather, Morgenroth, Lauren P., Henricson, Erik K., Duong, Tina, Hoffman, Eric P., Cnaan, Avital, McDonald, Craig M., Abresch, R.T., Cregan, M., Goude, E., Johnson, L., Han, J., Joyce, N., Nicorici, A., Reddy, D., Viswanathan, V., Chidambaranathan, C., Kumar, S., Lakshmi, V., Reddappa, P., McAdam, L., Biggar, D., Eliasoph, L., Hosaki, E., Harris, V., Lee, G., Mah, J., Chiu, A., Haig, T., Harris, M., Rincon, N., Sanchez, K., Walker, L., Tulinius, M., Alhander, A., Ekstrom, A., Gustafsson, A., Kroksmark, A., Sterky, U., Wahlgren, L., Thangarajh, M., Leshner, R., Tesi-Rocha, C., Birkmeier, M., Kaminski, S., Kornberg, A., Ryan, M., Carroll, K., DeValle, K., Kennedy, R., Rodriguez, V., Villano, D., Nevo, Y., Adani, R., Chen-Joseph, L., Daana, M., Leve, A. Bar, Panteleyev-Yitshak, V., Simchovitz, E., Yaffe, D., Dubrovsky, A., Andreone, L., Bonaudo, F., Corderi, J., Mesa, L., Marco, P., Levi, L., Clemens, P., Abdel-Hamid, H., Bendixen, R., Bise, C., Craig, A., Karnavas, K., Matthews, C., Niizawa, G., Smith, A., Weimer, J., Connolly, A., Pestronk, A., Florence, J., Christenson, T., Golumbak, P., Malkus, B., Renna, R., Schierbecker, J., Seiner, C., Wulf, C., Teasley, J., Blair, S., Grillo, B., Monasterio, E., Bertorini, T., Igarashi, M., Barrett-Adair, M., Carter, K., Clift, J., Gatlin, B., Holloway, J., Young, R.

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