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P314: Long-term safety and sustained functional benefit in patients with DMD 4 years post-treatment with delandistrogene moxeparvovec: A phase 1/2a study* 由 Erin O’Rourke, Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman, Linda P. Lowes, Natalie F. Reash, Megan Iammarino, Lindsay Alfano, Sarah Lewis, Kathleen Church, Richard Shell, Rachael A. Potter, Danielle Griffin, Mark J. Hogan, Shufang Wang, Stefanie Mason, Eddie Darton, Louise Rodino-Klapac
出版 2023-01-01
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Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA! 由 Amy Bartlett, Stephen J. Kolb, Allison Kingsley, Kathryn J. Swoboda, Sandra P. Reyna, Ai Sakonju, Basil T. Darras, Richard Shell, Nancy Kuntz, Diana Castro, Susan T. Iannaccone, Julie Parsons, Anne M. Connolly, Claudia A. Chiriboga, Craig McDonald, W. Bryan Burnette, Klaus Werner, Mathula Thangarajh, Perry B. Shieh, Erika Finanger, Christopher S. Coffey, Jon W. Yankey, Merit E. Cudkowicz, Michelle M. McGovern, D. Elizabeth McNeil, W. David Arnold, John T. Kissel
出版 2018-09-01
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