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Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

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.

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.

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

Erik Landfeldt, Joel Iff, Erik Henricson

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

Craig Campbell, Elaine McColl, Michael P McDermott, William B Martens, Michela Guglieri, Robert C Griggs

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, Nancy L Kuntz, Erica Koenig, Lilly East, Sameer Upadhyay, Baoguang Han, Perry B Shieh

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

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

Outcome Measures for COL6 and LAMA2-Related Dystrophies.

Abigail N Schwaede, Nancy L Kuntz

Pathogenic Variants in the Myosin Chaperone UNC-45B Cause Progressive Myopathy with Eccentric Cores.

Sandra Donkervoort, Carl E Kutzner, Ying Hu, Xavière Lornage, John Rendu, Tanya Stojkovic, Jonathan Baets, Sarah B Neuhaus, Jantima Tanboon, Reza Maroofian, Véronique Bolduc, Magdalena Mroczek, Stefan Conijn, Nancy L Kuntz, Ana Töpf, Soledad Monges, Fabiana Lubieniecki, Riley M McCarty, Katherine R Chao, Serena Governali, Johann Böhm, Kanokwan Boonyapisit, Edoardo Malfatti, Tumtip Sangruchi, Iren Horkayne-Szakaly, Carola Hedberg-Oldfors, Stephanie Efthymiou, Satoru Noguchi, Sarah Djeddi, Aritoshi Iida, Gabriella di Rosa, Chiara Fiorillo, Vincenzo Salpietro, Niklas Darin, Julien Fauré, Henry Houlden, Anders Oldfors, Ichizo Nishino, Willem de Ridder, Volker Straub, Wojciech Pokrzywa, Jocelyn Laporte, A Reghan Foley, Norma B Romero, Coen Ottenheijm, Thorsten Hoppe, Carsten G Bönnemann