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المؤلفون: Natalie F. Reash, Linda Lowes, Richard Shell, Samiah Al-Zaidy, Markus McColly, Aaron Kleyn, Lindsay N. Alfano, Matthew N. Meriggioli, Kathleen Church, Jerry R. Mendell, M. Iammarino, Kelly J. Lehman
المصدر: JAMA Neurol
مصطلحات موضوعية: Male, Pediatrics, medicine.medical_specialty, Recombinant Fusion Proteins, Phases of clinical research, Spinal Muscular Atrophies of Childhood, 03 medical and health sciences, 0302 clinical medicine, medicine, Humans, 030212 general & internal medicine, Adverse effect, Child, Original Investigation, Biological Products, Respiratory distress, business.industry, Incidence (epidemiology), Environmental air flow, Genetic Therapy, Discontinuation, Treatment Outcome, Child, Preschool, Cohort, Nusinersen, Female, Neurology (clinical), business, 030217 neurology & neurosurgery, Follow-Up Studies
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8eedc9fa05a91726b91e26b912220453
https://europepmc.org/articles/PMC8129901/ -
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المؤلفون: Giovanni Baranello, Haojun Ouyang, Arseniy Lavrov, Matthew N. Meriggioli, Riccardo Masson, Eugenio Mercuri, Giuseppe Vita, Claudio Bruno, Uwe Ernst, Volker Straub, James L’Italien, Douglas E. Feltner, Francesco Muntoni, Douglas M. Sproule, Stefania Corti, Susan Williamson, Elaine Kernbauer
المصدر: Neuropediatrics.
مصطلحات موضوعية: medicine.medical_specialty, business.industry, Gene replacement therapy, medicine, Urology, In patient, Spinal muscular atrophy, medicine.disease, business
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::2f93a701ae4c845014ea5d7a23fa647a
https://doi.org/10.1055/s-0039-1698171 -
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المؤلفون: Mark B. Bromberg, Miriam Freimer, Nizar Chahin, Laurie Gutmann, Mohammad Salajegheh, Kelly G. Gwathmey, Tyler A. Webb, David P. Richman, Aziz Shaibani, Guillermo Solorzano, Elliot L. Dimberg, Janice M. Massey, Rabi Tawil, James Gilchrist, Michael Benatar, Jeffrey A. Allen, Anthony J. Windebank, Summer Gibson, William J. Litchy, Yuebing Li, Amanda C. Guidon, James A. Russell, Vern C. Juel, William S. David, Shafeeq Ladha, Tahseen Mozaffar, Shawn J. Bird, David Saperstein, Chafic Karam, Noah Kolb, Gordon Smith, Gil I. Wolfe, W. David Arnold, Nicholas E. Johnson, Eric L. Logigian, John C. Kincaid, Duygu Selcen, Annabel K. Wang, Matthew N. Meriggioli, Andrew G. Engel, Pariwat Thaisetthawatkul, Lyle Ostrow, Yuen T. So, Jau Shin Lou, Michael K. Hehir, Eric J. Sorenson, P. James B. Dyck, George Sachs, Julie Khoury, Namita Goyal, Jeffrey T. Guptill, Jinny Tavee, Robert M. Pascuzzi, Jeffrey A. Cohen, Michael D. Weiss, Ted M. Burns, Yadollah Harati, Peter D. Donofrio, Jayashri Srinivasen, Perry B. Shieh, Daniel G Larriviere, Mark A. Ferrante, Sidney M. Gospe, Kathleen D. Kennelly, John T. Kissel, Clifton L. Gooch, Carlayne E. Jackson, Dmitri Gorelov, Nicholas Silvestri, Katherine Ruzhansky, Daniel J L Macgowen, Joon Shik Moon, Jonathan Goldstein, Robert G. Miller, Devon I. Rubin, Karissa L. Gable, Richard J. Barohn, Charles A. Thornton, Emma Ciafaloni, C. Michel Harper, Sarah M. Jones, Ricardo A. Maselli, J. Rob Singleton, Michelle M Mauermann, Brian A. Crum, James F. Howard, Erik R. Ensrud, Sami Khella, Mark A. Ross, Lisa D. Hobson-Webb, Sharon P. Nations, Stephen N. Scelsa, Katherine D. Mathews, Henry J. Kaminski, Andrea M. Corse, Amanda Peltier, Anthony A. Amato, Richard A. Lewis, Steven Vernino, Richard Nowak, Eduardo A De Sousa, Ludwig Gutmann, Benn E. Smith, Brent P. Goodman, David Lacomis, Jaya Trivedi
المصدر: Muscle & Nerve. 53:165-168
مصطلحات موضوعية: medicine.medical_specialty, Physiology, business.industry, Potassium channel blocker, medicine.disease, Orphan drug, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Physiology (medical), Orphan Drug Production, Environmental health, Neuromuscular junction disease, medicine, 030212 general & internal medicine, Neurology (clinical), Intensive care medicine, business, 030217 neurology & neurosurgery, medicine.drug
