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

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O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD* by Erin O’Rourke, Crystal Proud, Craig Zaidman, Perry B. Shieh, Craig McDonald, John W. Day, Stefanie Mason, Maitea Guridi, Lixin Han, Lixi Yu, Carol Reid, Eddie Darton, Christoph Wandel, James Richardson, Jyoti Malhotra, Teji Singh, Louise Rodino-Klapac, Jerry Mendell

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A randomized, double-blind, placebo-controlled phase II trial to explore the effects of a GABAA-α5 NAM (basmisanil) on intellectual disability associated with Down syndrome by Celia Goeldner, Priya S. Kishnani, Brian G. Skotko, Julian Lirio Casero, Joerg F. Hipp, Michael Derks, Maria-Clemencia Hernandez, Omar Khwaja, Sian Lennon-Chrimes, Jana Noeldeke, Sabine Pellicer, Lisa Squassante, Jeannie Visootsak, Christoph Wandel, Paulo Fontoura, Xavier Liogier d’Ardhuy, Clematis Study Group

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Basmisanil, a highly selective GABAA-α5 negative allosteric modulator: preclinical pharmacology and demonstration of functional target engagement in man by Joerg F. Hipp, Frederic Knoflach, Robert Comley, Theresa M. Ballard, Michael Honer, Gerhard Trube, Rodolfo Gasser, Eric Prinssen, Tanya L. Wallace, Andreas Rothfuss, Henner Knust, Sian Lennon-Chrimes, Michael Derks, Darren Bentley, Lisa Squassante, Stephane Nave, Jana Nöldeke, Christoph Wandel, Andrew W. Thomas, Maria-Clemencia Hernandez

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