检索结果 - Alexander P. Murphy
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Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy 由 Jerry R. Mendell, Crystal M. Proud, Craig M. Zaidman, Stefanie Mason, Eddie Darton, Shufang Wang, Christoph Wandel, Alexander P. Murphy, Eugenio Mercuri, Francesco Muntoni, Craig M. McDonald
出版 2024Artigo -
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Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy 由 Rachael A. Potter, Ida Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino‐Klapac
出版 2025Artigo -
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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1‐Year Interim Results from Study <scp>SRP</scp>‐9001‐10... 由 Craig M. Zaidman, Crystal M. Proud, Craig M. McDonald, Kelly J. Lehman, Natalie L. Goedeker, Stefanie Mason, Alexander P. Murphy, Maitea Guridi, Shufang Wang, Carol Reid, Eddie Darton, Christoph Wandel, Sarah Lewis, Jyoti Malhotra, Danielle A. Griffin, Rachael A. Potter, L. Rodino-Klapac, Jerry R. Mendell
出版 2023Artigo -
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229th ENMC international workshop: Limb girdle muscular dystrophies – Nomenclature and reformed classification Naarden, the Netherlands, 17–19 March 2017 由 Volker Straub, Alexander P. Murphy, Bjarne Udd, C. Angelini, Ségolène Aymé, Carsten Bönneman, Marjolein Visser, Ada Hamosh, Laura Jacobs, Nina Khizanishvili, Madelon Kroneman, Pascal Laflorêt, A. St. J. Murphy, Vincenzo Nigro, Laura Rufibach, Anna Sárközy, Shaun Swanepoel, Ivan Torrente, Bjarne Udd, Andoni Urtizberea, John Vissing, Maggie C. Walter
出版 2018Artigo -
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AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial 由 Jerry R. Mendell, Francesco Muntoni, Craig M. McDonald, Eugenio Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon‐Astudillo, A. Nascimento, Crystal M. Proud, Ulrike Schara‐Schmidt, Aravindhan Veerapandiyan, Craig M. Zaidman, Maitea Guridi, Alexander P. Murphy, Carol Reid, Christoph Wandel, Damon R. Asher, Eddie Darton, Stefanie Mason, Rachael A. Potter, Teji Singh, Wenfei Zhang, Paulo Fontoura, Jacob Elkins, Louise R. Rodino‐Klapac
出版 2024Artigo
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Biology
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Limb-girdle muscular dystrophy
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