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Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

International Consensus Guidance for Management of Myasthenia Gravis

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

Medical management of muscle weakness in Duchenne muscular dystrophy

Sarah R. Rivera, Anne M. Connolly, Basil T. Darras, Carolina Tesi Rocha, Chamindra G. Konersman, Craig M. McDonald, Craig M. Zaidman, Emma Ciafaloni, Gyula Acsadi, Hoda Z. Abdel-Hamid, John Brandsema, Julie A. Parsons, Katherine D. Mathews, Nancy L. Kuntz, Russell J. Butterfield, Sumit K. Jhamb, Susan T. Iannaccone

Combination molecular therapies for type 1 spinal muscular atrophy.

Yohei Harada, Amit Agarwal, Aravindhan Veerapandiyan, Christine J. DiDonato, Galia Napchan-Pomerantz, Kapil Arya, Masahisa Katsuno, Nancy L. Kuntz, Vamshi K. Rao, Vikki Stefans

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

Edward C. Smith, Adrienne Arrieta, Benjamin D. Schwartz, Craig M. McDonald, Diana Castro, Eric P. Hoffman, Heather Gordish-Dressman, Jean K. Mah, Jesse M. Damsker, Laura Hagerty, Laurel J. Mengle-Gaw, Lauren P. Morgenroth, Laurie Kerchner, Laurie S. Conklin, Mar Tulinius, Mark Jaros, Maya Shimony, Michela Guglieri, Monique M. Ryan, Nancy L. Kuntz, Paula R. Clemens, Phil Shale, Richard Webster, Richard S. Finkel, Utkarsh J. Dang, Yoram Nevo

The CINRG Becker Natural History Study: Baseline characteristics

Paula R. Clemens, Amy Harper, Andrea Smith D'Alessandro, Anne Connolly, Avital Cnaan, Barry Byrne, Christopher Spurney, Craig M. McDonald, Edward Smith, Elena Bravver, Erik K. Henricson, Gabriela Niizawa, Georgios Manousakis, Heather Gordish-Dressman, Jean K. Mah, Jia Feng, Julaine Florence, Ksenija Gorni, Lauren P. Morgenroth, Mathula Thangarajh, Matthew Wicklund, Michela Guglieri, Nancy Kuntz, Saila Upadhyayula, Susan Iannaccone, Tulio Bertorini

Difficulties classifying myasthenia gravis in the pediatric surgical literature

Re: “Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy” by Wilson and Flotte

Spinal muscular atrophy

Spinal muscular atrophy care in the COVID-19 pandemic era.

Aravindhan Veerapandiyan, Anne M. Connolly, Diana Castro, Edward C. Smith, Emma Ciafaloni, Eugenio Mercuri, John F. Brandsema, Julie A. Parsons, Kapil Arya, Katherine D. Mathews, Laurent Servais, Nancy Kuntz, Richard S. Finkel, Russell J. Butterfield, Vamshi K. Rao