Clinical potential of microdystrophin as a surrogate endpoint von Boehler, Jessica F, Brown, Kristy J, Beatka, Margaret, Gonzalez, J Patrick, Donisa Dreghici, Roxana, Soustek-Kramer, Meghan, McGonigle, Sharon, Ganot, Annie, Palmer, Timothy, Lowie, Caitlin, Chamberlain, Jeffrey S., Lawlor, Michael W, Morris, Carl A

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Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint von Servais, Laurent, Strijbos, Paul, Poleur, Margaux, Mirea, Andrada, Butoianu, Nina, Sansone, Valeria A., Vuillerot, Carole, Schara-Schmidt, Ulrike, Scoto, Mariacristina, Seferian, Andreea M., Previtali, Stefano C., Tulinius, Már, Nascimento, Andrés, Furlong, Pat, Singh, Teji, Dreghici, Roxana Donisa, Goemans, Nathalie, Mercuri, Eugenio, Straub, Volker, Ormazabal, Maitea Guridi, Braid, Jessica, Muntoni, Francesco, Tricot, Alexis, Annoussamy, Mélanie, Eggenspieler, Damien

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Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy von Cope, Heidi, Fischer, Ryan, Heslop, Emma, McNiff, Megan, Johnson, Alexandra, Camino, Eric, Denger, Brian, Armstrong, Niki, Thakrar, Sejal, Bateman-House, Alison, Beaverson, Katherine L, Woollacott, Ione O C, Phillips, Dawn, Fernandez, Vivian, Ganot, Annie, Donisa-Dreghici, Roxana, Mansfield, Carol, Peay, Holly

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