Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management von Bushby, Katharine, MD, Finkel, Richard, MD, Birnkrant, David J, MD, Case, Laura E, DPT, Clemens, Paula R, MD, Cripe, Linda, MD, Kaul, Ajay, MD, Kinnett, Kathi, MSN, McDonald, Craig, MD, Pandya, Shree, PT, Poysky, James, PhD, Shapiro, Frederic, MD, Tomezsko, Jean, PhD, Constantin, Carolyn, PhD

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Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care von Bushby, Katharine, MD, Finkel, Richard, MD, Birnkrant, David J, MD, Case, Laura E, DPT, Clemens, Paula R, MD, Cripe, Linda, MD, Kaul, Ajay, MD, Kinnett, Kathi, MSN, McDonald, Craig, MD, Pandya, Shree, PT, Poysky, James, PhD, Shapiro, Frederic, MD, Tomezsko, Jean, PhD, Constantin, Carolyn, PhD

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Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy von ESCOLAR, D. M, HACHE, L. P, PESTRONK, A, RYAN, M. M, MONASTERIO, E, DAY, J. W, ZIMMERMAN, A, ARRIETA, A, HENRICSON, E, MAYHEW, J, FLORENCE, J, HU, F, CLEMENS, P. R, CONNOLLY, A. M, CNAAN, A, MCDONALD, C. M, VISWANATHAN, V, KORNBERG, A. J, BERTORINI, T. E, NEVO, Y, LOTZE, T

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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug von Conklin, Laurie S., Damsker, Jesse M., Hoffman, Eric P., Jusko, William J., Mavroudis, Panteleimon D., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Mah, Jean K., Guglieri, Michela, Nevo, Yoram, Kuntz, Nancy, McDonald, Craig M., Tulinius, Mar, Ryan, Monique M., Webster, Richard, Castro, Diana, Finkel, Richard S., Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Jaros, Mark, Shale, Phil, McCall, John M., Hathout, Yetrib, Nagaraju, Kanneboyina, van den Anker, John, Ward, Leanne M., Ahmet, Alexandra, Cornish, Michaelyn R., Clemens, Paula R.

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Identification of a Neuronal Nitric Oxide Synthase in Isolated Cardiac Mitochondria Using Electrochemical Detection von Kanai, Anthony J., Pearce, Linda L., Clemens, Paula R., Birder, Lori A., VanBibber, Michelle M., Choi, So-Young, de Groat, William C., Peterson, Jim

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CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy von Escolar, Diana M., Buyse, Gunnar, Henricson, Erik, Leshner, Robert, Florence, Julaine, Mayhew, Jill, Tesi-Rocha, Carolina, Gorni, Ksenija, Pasquali, Livia, Patel, Kantilal M., McCarter, Robert, Huang, Jennifer, Mayhew, Thomas, Bertorini, Tulio, Carlo, Jose, Connolly, Anne M., Clemens, Paula R., Goemans, Nathalie, Iannaccone, Susan T., Igarashi, Masanori, Nevo, Yoram, Pestronk, Alan, Subramony, S. H., Vedanarayanan, V. V., Wessel, Henry

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Comparison of high-capacity and first-generation adenoviral vector gene delivery to murine muscle in utero von BILBAO, R, REAY, D. P, WU, E, ZHENG, H, BIERMANN, V, KOCHANEK, S, CLEMENS, P. R

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Immune Response to Full-Length Dystrophin Delivered to Dmd Muscle by a High-Capacity Adenoviral Vector von Gilchrist, Soyoung C., Ontell, Martin P., Kochanek, Stefan, Clemens, Paula R.

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Sustained Muscle Expression of Dystrophin from a High-Capacity Adenoviral Vector with Systemic Gene Transfer of T Cell Costimulatory Blockade von Jiang, Zhilong, Schiedner, Gudrun, van Rooijen, Nico, Liu, Chau-Ching, Kochanek, Stefan, Clemens, Paula R

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Muscle glycogenosis with low phosphorylase kinase activity: mutations in PHKA1, PHKG1 or six other candidate genes explain only a minority of cases von Burwinkel, Barbara, Hu, Bin, Schroers, Anja, Clemens, Paula R, Moses, Shimon W, Shin, Yoon S, Pongratz, Dieter, Vorgerd, Matthias, Kilimann, Manfred W

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Patterns of gene expression from in utero delivery of adenoviral-associated vector serotype 1 von BILBAO, Roberto, REAY, Daniel P, JUAN LI, XIAO XIAO, CLEMENS, Paula R

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Carrier detection and prenatal diagnosis in Duchenne and Becker muscular dystrophy families, using dinucleotide repeat polymorphisms von CLEMENS, P. R, FENWICK, R. G, CHAMBERLAIN, J. S, GIBBS, R. A, DE ANDRADE, M, CHAKRABORTY, R, CASKEY, C. T

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DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle von Chen, H H, Mack, L M, Choi, S Y, Ontell, M, Kochanek, S, Clemens, P R

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Chronic eosinophilic perimyositis with persistent myalgias von Zivkovic, Sasa A., Lacomis, David, Clemens, Paula R.

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Binding of adenoviral fiber knob to the coxsackievirus-adenovirus receptor is crucial for transduction of fetal muscle von BILBAO, Roberto, SRINIVASAN, Suchitra, REAY, Daniel, GOLDBERG, Laura, HUGHES, Tiffany, ROELVINK, Peter W, EINFELD, David A, WICKHAM, Thomas J, CLEMENS, Paula R

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Pentanucleotide repeat length polymorphism at the human CD4 locus von EDWARDS, M. C, CLEMENS, P. R, TRISTAN, M, PIZZUTI, A, GIBBS, R. A

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Efficacy and Safety of Vamorolone vs Placebo and Prednisone among Boys with Duchenne Muscular Dystrophy: A Randomized Clinical Trial von Guglieri, M, Clemens, PR, Perlman, SJ, Smith, EC, Horrocks, I, Finkel, RS, Mah, JK, Deconinck, N, Goemans, N, Haberlova, J, Straub, V, Mengle-Gaw, LJ, Schwartz, BD, Harper, AD, Shieh, PB, De Waele, L, Castro, D, Yang, ML, Ryan, MM, McDonald, CM, Tulinius, M, Webster, R, McMillan, HJ, Kuntz, NL, Rao, VK, Baranello, G, Spinty, S, Childs, AM, Sbrocchi, AM, Selby, KA, Monduy, M, Nevo, Y, Vilchez-Padilla, JJ, Nascimento-Osorio, A, Niks, EH, De Groot, IJM, Katsalouli, M, James, MK, Van Den Anker, J, Damsker, JM, Ahmet, A, Ward, LM, Jaros, M, Shale, P, Dang, UJ, Hoffman, EP

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Adult phosphorylase b kinase deficiency von Clemens, Paula R., Yamamoto, Masahiko, Engel, Andrew G.

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Recombinant truncated dystrophin minigenes: construction, expression, and adenoviral delivery von Clemens, P R, Krause, T L, Chan, S, Korb, K E, Graham, F L, Caskey, C T

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Dopamine Receptor D3 Agonist (Pramipexole) Abolishes Morphine‐Induced Cardiac Fibrosis in Mice von Gaweda, Gabriel G., Iyer, Rugmani P., Shaver, Patti R., Grilo, Gabriel A., Dinkins, Mai‐Lynne, Stoffel, Hamilton J., Clemens, Stefan, Castro Brás, Lisandra E.

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