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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
Published 2023-01-01
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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint by Laurent Servais, Paul Strijbos, Margaux Poleur, Andrada Mirea, Nina Butoianu, Valeria A. Sansone, Carole Vuillerot, Ulrike Schara-Schmidt, Mariacristina Scoto, Andreea M. Seferian, Stefano C. Previtali, Már Tulinius, Andrés Nascimento, Pat Furlong, Teji Singh, Roxana Donisa Dreghici, Nathalie Goemans, Eugenio Mercuri, Volker Straub, Maitea Guridi Ormazabal, Jessica Braid, Francesco Muntoni, Alexis Tricot, Mélanie Annoussamy, Damien Eggenspieler
Published 2024-11-01
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Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscu... by Jerry R. Mendell, Jerry R. Mendell, Jerry R. Mendell, Perry B. Shieh, Craig M. McDonald, Zarife Sahenk, Zarife Sahenk, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Linda P. Lowes, Natalie F. Reash, Megan A. Iammarino, Lindsay N. Alfano, Brenna Sabo, Jeremy D. Woods, Christy L. Skura, Howard C. Mao, Loretta A. Staudt, Danielle A. Griffin, Sarah Lewis, Shufang Wang, Rachael A. Potter, Teji Singh, Louise R. Rodino-Klapac
Published 2023-07-01
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