Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study by Bello, Luca, Kesari, Akanchha, Gordish-Dressman, Heather, Cnaan, Avital, Morgenroth, Lauren P., Punetha, Jaya, Duong, Tina, Henricson, Erik K., Pegoraro, Elena, McDonald, Craig M., Hoffman, Eric P.

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Engaging Participants in Rare Disease Research: A Qualitative Study of Duchenne Muscular Dystrophy by Bendixen, Roxanna M., PhD, OTR/L, Morgenroth, Lauren P., MS, CGC, Clinard, Kristin L., MS, CGC

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Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study by Boca, Simina M, Nishida, Maki, Harris, Michael, Rao, Shruti, Cheema, Amrita K, Gill, Kirandeep, Seol, Haeri, Morgenroth, Lauren P, Henricson, Erik, McDonald, Craig, Mah, Jean K, Clemens, Paula R, Hoffman, Eric P, Hathout, Yetrib, Madhavan, Subha

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Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children by Hathout, Yetrib, Conklin, Laurie S., Seol, Haeri, Gordish-Dressman, Heather, Brown, Kristy J., Morgenroth, Lauren P., Nagaraju, Kanneboyina, Heier, Christopher R., Damsker, Jesse M., van den Anker, John N., Henricson, Erik, Clemens, Paula R., Mah, Jean K., McDonald, Craig, Hoffman, Eric P.

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Correction: Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study by Boca, Simina M, Nishida, Maki, Harris, Michael, Rao, Shruti, Cheema, Amrita K, Gill, Kirandeep, Wang, Difei, An, Lin, Gauba, Robinder, Seol, Haeri, Morgenroth, Lauren P, Henricson, Erik, McDonald, Craig, Mah, Jean K, Clemens, Paula R, Hoffman, Eric P, Hathout, Yetrib, Madhavan, Subha

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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 by 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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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study by Smith, Edward C., Conklin, Laurie S., Hoffman, Eric P., Clemens, Paula R., Mah, Jean K., Finkel, Richard S., Guglieri, Michela, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Castro, Diana, Kuntz, Nancy L., Kerchner, Laurie, Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Jaros, Mark, Shale, Phil, Gordish-Dressman, Heather, Hagerty, Laura, Dang, Utkarsh J., Damsker, Jesse M., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., McDonald, Craig M.

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Neurology Care, Diagnostics, and Emerging Therapies of the Patient With Duchenne Muscular Dystrophy by Leigh, Fawn, Ferlini, Alessandra, Biggar, Doug, Bushby, Katharine, Finkel, Richard, Morgenroth, Lauren P, Wagner, Kathryn R

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Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim... by Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.

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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug by 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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Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022)... by Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.

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The CINRG Becker Natural History Study: Baseline characteristics by Clemens, Paula R., Niizawa, Gabriela, Feng, Jia, Florence, Julaine, DʼAlessandro, Andrea Smith, Morgenroth, Lauren P., Gorni, Ksenija, Guglieri, Michela, Connolly, Anne, Wicklund, Matthew, Bertorini, Tulio, Mah, Jean K., Thangarajh, Mathula, Smith, Edward, Kuntz, Nancy, McDonald, Craig M., Henricson, Erik K., Upadhyayula, Saila, Byrne, Barry, Manousakis, Georgios, Harper, Amy, Bravver, Elena, Iannaccone, Susan, Spurney, Christopher, Cnaan, Avital, Gordish‐Dressman, Heather

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TCTEX1D1 is a genetic modifier of disease progression in Duchenne muscular dystrophy by Spitali, Pietro, Zaharieva, Irina, Bohringer, Stefan, Hiller, Monika, Chaouch, Amina, Roos, Andreas, Scotton, Chiara, Claustres, Mireille, Bello, Luca, McDonald, Craig M, Hoffman, Eric P, Koeks, Zaida, Eka Suchiman, H, Cirak, Sebahattin, Scoto, Mariacristina, Reza, Mojgan, 't Hoen, Peter A C, Niks, Erik H, Tuffery-Giraud, Sylvie, Lochmüller, Hanns, Ferlini, Alessandra, Muntoni, Francesco, Aartsma-Rus, Annemieke

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Evidence for ACTN3 as a genetic modifier of Duchenne muscular dystrophy by Hogarth, Marshall W., Houweling, Peter J., Thomas, Kristen C., Gordish-Dressman, Heather, Bello, Luca, Pegoraro, Elena, Hoffman, Eric P., Head, Stewart I., North, Kathryn N.

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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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Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy by Landfeldt, Erik, Iff, Joel, Henricson, Erik, Vishwanathan, V., Chidambaranathan, S., Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Tulinius, Mar, Cnaan, Avital, Morgenroth, Lauren P., Leshner, Robert, TesiRocha, 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, Bertorini, 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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Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy by Conrado, Daniela J., Larkindale, Jane, Berg, Alexander, Hill, Micki, Burton, Jackson, Abrams, Keith R., Abresch, Richard T., Bronson, Abby, Chapman, Douglass, Crowther, Michael, Duong, Tina, Gordish-Dressman, Heather, Harnisch, Lutz, Henricson, Erik, Kim, Sarah, McDonald, Craig M., Schmidt, Stephan, Vong, Camille, Wang, Xiaoxing, Wong, Brenda L., Yong, Florence, Romero, Klaus

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Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients by McDonald, Craig M, Muntoni, Francesco, Penematsa, Vinay, Jiang, Joel, Kristensen, Allan, Bibbiani, Francesco, Goodwin, Elizabeth, Gordish-Dressman, Heather, Morgenroth, Lauren, Werner, Christian, Li, James, Able, Richard, Trifillis, Panayiota, Tulinius, Már

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Genetic modifiers of respiratory function in Duchenne muscular dystrophy by Bello, Luca, D’Angelo, Grazia, Villa, Matteo, Fusto, Aurora, Vianello, Sara, Merlo, Beatrice, Sabbatini, Daniele, Barp, Andrea, Gandossini, Sandra, Magri, Francesca, Comi, Giacomo P., Pedemonte, Marina, Tacchetti, Paola, Lanzillotta, Valentina, Trucco, Federica, D’Amico, Adele, Bertini, Enrico, Astrea, Guja, Politano, Luisa, Masson, Riccardo, Baranello, Giovanni, Albamonte, Emilio, De Mattia, Elisa, Rao, Fabrizio, Sansone, Valeria A., Previtali, Stefano, Messina, Sonia, Vita, Gian Luca, Berardinelli, Angela, Mongini, Tiziana, Pini, Antonella, Pane, Marika, Mercuri, Eugenio, Vianello, Andrea, Bruno, Claudio, Hoffman, Eric P., Morgenroth, Lauren, Gordish‐Dressman, Heather, McDonald, Craig M., Pegoraro, Elena

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