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1دورية أكاديمية
المؤلفون: Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group
المصدر: Neurology and Therapy, Vol 12, Iss 2, Pp 543-557 (2023)
مصطلحات موضوعية: Evrysdi, Pharmacodynamics, Risdiplam, Safety, Spinal muscular atrophy, Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
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2دورية أكاديمية
المؤلفون: Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group
المصدر: Neurology and Therapy, Vol 12, Iss 5, Pp 1799-1801 (2023)
مصطلحات موضوعية: Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
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3دورية أكاديمية
المؤلفون: Claude Cances, Dmitry Vlodavets, Giacomo Pietro Comi, Riccardo Masson, Maria Mazurkiewicz-Bełdzińska, Kayoko Saito, Edmar Zanoteli, Angela Dodman, Muna El-Khairi, Ksenija Gorni, Isaac Gravestock, Janine Hoffart, Renata S. Scalco, Basil T. Darras, the ANCHOVY Working Group
المصدر: Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-11 (2022)
مصطلحات موضوعية: ANCHOVY, FIREFISH, SMA natural history, Type 1 SMA, Spinal muscular atrophy, Medicine
وصف الملف: electronic resource
العلاقة: https://doaj.org/toc/1750-1172Test
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4دورية أكاديمية
المؤلفون: Mélanie Annoussamy, Andreea M. Seferian, Aurore Daron, Yann Péréon, Claude Cances, Carole Vuillerot, Liesbeth De Waele, Vincent Laugel, Ulrike Schara, Teresa Gidaro, Charlotte Lilien, Jean‐Yves Hogrel, Pierre Carlier, Emmanuel Fournier, Linda Lowes, Ksenija Gorni, Myriam Ly‐Le Moal, Nicole Hellbach, Timothy Seabrook, Christian Czech, Ricardo Hermosilla, Laurent Servais, the NatHis‐SMA study group
المصدر: Annals of Clinical and Translational Neurology, Vol 8, Iss 2, Pp 359-373 (2021)
مصطلحات موضوعية: Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571, Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
العلاقة: https://doaj.org/toc/2328-9503Test
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5دورية أكاديمية
المؤلفون: Dylan Trundell, Stephanie Le Scouiller, Ksenija Gorni, Timothy Seabrook, Carole Vuillerot, the SMA MFM Study Group
المصدر: Neurology and Therapy, Vol 9, Iss 2, Pp 575-584 (2020)
مصطلحات موضوعية: 32-item Motor Function Measure, Clinician-reported outcome, Reliability, Spinal muscular atrophy, Validity, Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
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6دورية أكاديمية
المؤلفون: Tina Duong, Hannah Staunton, Jessica Braid, Aurelie Barriere, Ben Trzaskoma, Ling Gao, Tom Willgoss, Rosangel Cruz, Nicole Gusset, Ksenija Gorni, Sharan Randhawa, Lida Yang, Carole Vuillerot
المصدر: Frontiers in Neurology, Vol 12 (2022)
مصطلحات موضوعية: 32-item Motor Function Measure (MFM32), spinal muscular atrophy, meaningful change, qualitative interviews, online survey, anchor-based methods, Neurology. Diseases of the nervous system, RC346-429
وصف الملف: electronic resource
العلاقة: https://www.frontiersin.org/articles/10.3389/fneur.2021.770423/fullTest; https://doaj.org/toc/1664-2295Test
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7دورية أكاديمية
المصدر: PLoS ONE, Vol 15, Iss 9, p e0238786 (2020)
وصف الملف: electronic resource
العلاقة: https://doaj.org/toc/1932-6203Test
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8دورية أكاديمية
المؤلفون: Andrea Barp, Luca Bello, Luisa Politano, Paola Melacini, Chiara Calore, Angela Polo, Sara Vianello, Gianni Sorarù, Claudio Semplicini, Boris Pantic, Antonella Taglia, Ester Picillo, Francesca Magri, Ksenija Gorni, Sonia Messina, Gian Luca Vita, Giuseppe Vita, Giacomo P Comi, Mario Ermani, Vincenzo Calvo, Corrado Angelini, Eric P Hoffman, Elena Pegoraro
المصدر: PLoS ONE, Vol 10, Iss 10, p e0141240 (2015)
وصف الملف: electronic resource
العلاقة: http://europepmc.org/articles/PMC4626372?pdf=renderTest; https://doaj.org/toc/1932-6203Test
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9
المؤلفون: Maggie C. Walter, Pascal Laforêt, W. Ludo van der Pol, Elena Pegoraro, Shahram Attarian, Bart Bartels, Ksenija Gorni, Nathalie Goemans, Nicole Gusset, Victoria Hodgkinson, Tim Hagenacker, Janbernd Kirschner, Andrea Klein, Anna Kostera-Pruszczyk, Hanns Lochmüller, Chiara Marini-Bettolo, Eugenio Mercuri, Robert Muni-Lofra, Laetitia Ouillade, Rosaline Quinlivan, Constantinos Papadopoulos, Hélène Prigent, Emmanuelle Salort-Campana, Valeria A Sansone, Rivka Smit, Piera Smeriglio, Simone Thiele, Ben Tichler, Peter Van den Bergh, Juan F Vazquez-Costa, John Vissing
المصدر: Neuromuscular Disorders. 33:511-522
مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Medizin, Neurology (clinical), Genetics (clinical)
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::358ba1ce701ca027f0bfab2def0d78b1Test
https://doi.org/10.1016/j.nmd.2023.03.011Test -
10مؤتمر
المؤلفون: Laurent, Servais, Giovanni, Baranello, Claudia, Chiriboga, Basil, Darras, Marianne, Gerber, Ksenija, Gorni, Birgit, Jaber, Tammy, McIver, Renata, Scalco, Eugenio, Mercuri
المصدر: Association of British Neurologists: Annual Meeting Abstracts 2023