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INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy

Development of a model‐based clinical trial simulation platform to optimize the design of clinical trials for Duchenne muscular dystrophy

Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model.

Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.

Health related quality of life in young, steroid-naïve boys with Duchenne muscular dystrophy.

Craig Campbell, Adnan Y. Manzur, Anne-Marie Childs, Ashutosh Kumar, Basil T. Darras, Craig M. McDonald, Ekkehard Wilichowski, Elaine McColl, Elena Pegoraro, Emma Ciafaloni, Erik K. Henricson, Federica Ricci, Gian Luca Vita, Giovanni Baranello, Giuseppe Vita, Helen Roper, Hugh J. McMillan, Iain Horrocks, Imelda Hughes, James F. Howard, Janbernd Kirschner, Jean K. Mah, Jeffrey Statland, Kevin M. Flanigan, Leslie Morrison, Lorenzo Maggi, Luca Bello, Maja von der Hagen, Mathula Thangarajh, Matthew Wicklund, Michael P. McDermott, Michela Guglieri, Monika Krzesniak-Swinarska, Nancy L. Kuntz, Perry B. Shieh, Peter Kang, Richard J. Barohn, Richard S. Finkel, Robert C. Griggs, Russell J. Butterfield, Stefan Spinty, Taeun Chang, Tiziana Mongini, Ulrike Schara-Schmidt, Volker Straub, W. Bryan Burnette, William B. Martens

Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

Erik Landfeldt, Alan Pestronk, Alberto Dubrovsky, Andrew Kornberg, Anne M. Connolly, Avital Cnaan, Carolina TesiRocha, Craig M. McDonald, Erik Henricson, Erik K. Henricson, Hanna Kolski, Hoda Abdel-Hamid, Jean Teasley, Jean K. Mah, Joel Iff, John B. Bodensteiner, John W. Day, Jose Carlo, Ksenija Gorni, Laura C. McAdam, Lauren P. Morgenroth, Mar Tulinius, Mathula Thangarajh, Monique Ryan, Nancy Kuntz, Nanette C. Joyce, Paula R. Clemens, Peter Karachunski, Richard Webster, Richard T. Abresch, Robert Leshner, S. Chidambaranathan, Sherilyn Driscoll, Timothy Lotze, Tina Duong, Tulio E. Bertorini, V. Vishwanathan, W. Douglas Biggar, Yoram Nevo

Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial

Kathryn R. Wagner, Baoguang Han, Erica Koenig, Lilly East, Nancy L. Kuntz, Perry B. Shieh, Sameer Upadhyay

Correction to: Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study.

Stacy A. Rudnicki, Ali A. Habib, Andrew A. Wolff, Angela Genge, Anne M. Connolly, Basil T. Darras, Bettina M. Cockroft, Carolina Tesi-Rocha, Craig Campbell, Fady I. Malik, Jean K. Mah, Jeffrey Statland, Jenny Wei, Jinsy A. Andrews, John W. Day, Kevin Felice, Lisa Meng, Maryam Oskoui, Nancy L. Kuntz, Nicholas E. Johnson, Perry B. Shieh, Richard S. Finkel, Tina Duong