Electrical impedance myography in duchenne muscular dystrophy and healthy controls: A multicenter study of reliability and validity by Zaidman, Craig M., Wang, Lucy L., Connolly, Anne M., Florence, Julaine, Wong, Brenda L., Parsons, Julie A., Apkon, Susan, Goyal, Namita, Williams, Eugene, Escolar, Diana, Rutkove, Seward B., Bohorquez, Jose L.

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Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis ty... by Malkus, Betsy C., Cohen, Jeanne M., Grant, Christina L., Scarpa, Maurizio, Sun, Angela, Wang, Raymond Y., Burton, Barbara K., Oussoren, Esmeralda, Martins, Esmeralda E., Durand, Consuelo, Chabrol, Brigitte, Ortiz, Lionel, Hetzer, Joel, Marsden, Deborah, Merritt, J. Lawrence, Giugliani, Roberto, Gonzalez-Meneses, Antonio

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Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in subjects with mucopolysaccharidosis type VII < 5 years old by Lau, Heather, Viskochil, David, Tanpaiboon, Pranoot, Lopez, Antonio Gonzalez-Meneses, Martins, Esmeralda, Taylor, Julie, Cimms, Tricia, Malkus, Betsy C., Chang, Ting, Zhang, Lin, Marsden, Deborah, Jurecka, Agnieszka

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The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduce... by Henricson, Erik K., Abresch, R. Ted, Cnaan, Avital, Hu, Fengming, Duong, Tina, Arrieta, Adrienne, Han, Jay, Escolar, Diana M., Florence, Julaine M., Clemens, Paula R., Hoffman, Eric P., McDonald, Craig M.

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Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy by Bello, Luca, Flanigan, Kevin M., Weiss, Robert B., Dunn, Diane M., Swoboda, Kathryn J., Gappmaier, Eduard, Howard, Michael T., Sampson, Jacinda B., Bromberg, Mark B., Butterfield, Russell, Kerr, Lynne, Pestronk, Alan, Florence, Julaine M., Connolly, Anne, Lopate, Glenn, Golumbek, Paul, Schierbecker, Jeanine, Malkus, Betsy, Renna, Renee, Siener, Catherine, Finkel, Richard S., Bonnemann, Carsten G., Medne, Livija, Glanzman, Allan M., Flickinger, Jean, Mendell, Jerry R., King, Wendy M., Lowes, Linda, Alfano, Lindsay, Mathews, Katherine D., Stephan, Carrie, Laubenthal, Karla, Baldwin, Kris, Wong, Brenda, Morehart, Paula, Meyer, Amy, Day, John W., Naughton, Cameron E., Margolis, Marcia, Spitali, Pietro, Aartsma-Rus, Annemieke, Muntoni, Francesco, Zaharieva, Irina, Ferlini, Alessandra, Mercuri, Eugenio, Tuffery-Giraud, Sylvie, Claustres, Mireille, Straub, Volker, Lochmüller, Hanns, Barp, Andrea, Vianello, Sara, Pegoraro, Elena, Punetha, Jaya, Gordish-Dressman, Heather, Giri, Mamta, McDonald, Craig M., Hoffman, Eric P., Cnaan, Avital, Abresch, Richard T., Henricson, Erik K., Morgenroth, Lauren P., Duong, Tina, Chidambaranathan, V. Viswanathan, Biggar, W. Douglas, McAdam, Laura C., Mah, Jean, Tulinius, Mar, Leshner, Robert, Rocha, Carolina Tesi, Thangarajh, Mathula, Kornberg, Andrew, Ryan, Monique, Nevo, Yoram, Dubrovsky, Alberto, Clemens, Paula R., Abdel-Hamid, Hoda, Connolly, Anne M., Teasley, Jean, Bertorini, Tulio E., North, Kathryn, Webster, Richard, Kolski, Hanna, Kuntz, Nancy, Driscoll, Sherilyn, Carlo, Jose, Gorni, Ksenija, Lotze, Timothy, Karachunski, Peter, Bodensteiner, John B.

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Cooperative international neuromuscular research group duchenne natural history study demonstrates insufficient diagnosis and treatment of cardiomyopathy in duchenne muscular dystr... by Spurney, Christopher, Shimizu, Reiko, Morgenroth, Lauren P., Kolski, Hanna, Gordish-Dressman, Heather, Clemens, Paula R.

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