Healthcare Utilization, Costs of Care, and Mortality Among Patients With Spinal Muscular Atrophy ‌ Hiangkiat Tan, Tao Gu, Er Chen, Rajeshwari Punekar, Perry B. Shieh

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The Last Mile: DSCSA Solution Through Blockchain Technology: Drug Tracking, Tracing, and Verification at the Last Mile of the Pharmaceutical Supply Chain with BRUINchain ‌ William Chien, Josenor de Jesus, Ben Taylor, Victor Dods, Leo Alekseyev, Diane Shoda, Perry B. Shieh

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Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status ‌ Craig M McDonald, Jessica R Marden, Perry B Shieh, Brenda L Wong, Henry Lane, Adina Zhang, Ha Nguyen, Molly Frean, Panayiota Trifillis, Karyn Koladicz, James Signorovitch

O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD* ‌ 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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Current insights in ultra-rare adenylosuccinate synthetase 1 myopathy – meeting report on the First Clinical and Scientific Conference. 3 June 2024, National Centre for Advancing T... ‌ Emma Rybalka, Hyung Jun Park, Atchayaram Nalini, Dipti Baskar, Kiran Polavarapu, Hacer Durmus, Yang Xia, Linlin Wan, Perry B. Shieh, Behzad Moghadaszadeh, Alan H. Beggs, David L. Mack, Alec S. T. Smith, Wendy Hanna-Rose, Hyder A. Jinnah, Cara A. Timpani, Min Shen, Jaymin Upadhyay, Jeffrey J. Brault, Matthew D. Hall, Naveen Baweja, Priyanka Kakkar

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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... ‌ 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

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Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA! ‌ 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

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A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial ‌ Rebecca A. Crow, Kimberly A. Hart, Michael P. McDermott, Rabi Tawil, William B. Martens, Barbara E. Herr, Elaine McColl, Jennifer Wilkinson, Janbernd Kirschner, Wendy M. King, Michele Eagle, Mary W. Brown, Deborah Hirtz, Hanns Lochmuller, Volker Straub, Emma Ciafaloni, Perry B. Shieh, Stefan Spinty, Anne-Marie Childs, Adnan Y. Manzur, Lucia Morandi, Russell J. Butterfield, Iain Horrocks, Helen Roper, Kevin M. Flanigan, Nancy L. Kuntz, Jean K. Mah, Leslie Morrison, Basil T. Darras, Maja von der Hagen, Ulrike Schara, Ekkehard Wilichowski, Tiziana Mongini, Craig M. McDonald, Giuseppe Vita, Richard J. Barohn, Richard S. Finkel, Matthew Wicklund, Hugh J. McMillan, Imelda Hughes, Elena Pegoraro, W. Bryan Burnette, James F. Howard, Mathula Thangarajh, Craig Campbell, Robert C. Griggs, Kate Bushby, Michela Guglieri

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