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A patient-centered qualitative evaluation of meaningful change on the NSAA and PUL in Duchenne Muscular Dystrophy by Aviva Gillman, Teofil Ciobanu, Louise Barrett, Evan W. Davies, Alexander P. Murphy, Alex Johnson, Jessica Mills, Phoebe Heinrich, Krystian Przydzial, Bethany Ewens, Gerrit Vandenberg, Stefan Cano, Anna Mayhew
Published 2025-03-01Get full text
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Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy by Rachael A. Potter, Ida H. 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
Published 2025-01-01Get full text
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