599PThe integration of PROMs and real-world data: a holistic approach to characterise disease burden and treatment impact in spinal muscular atrophy.
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| Title: | 599PThe integration of PROMs and real-world data: a holistic approach to characterise disease burden and treatment impact in spinal muscular atrophy. |
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| Authors: | Carver, A.1 (AUTHOR), Benesperi, G.1 (AUTHOR), Karkkainen, E.1 (AUTHOR), Moat, D.1 (AUTHOR), Page, J.1 (AUTHOR), Tanner, S.1 (AUTHOR), Cavalcante, E.2 (AUTHOR), Fitzpatrick, C.2 (AUTHOR), Joefield, T.2 (AUTHOR), Adcock, K.3 (AUTHOR), Farrugia, M.4 (AUTHOR), Ilyashenko, G.5 (AUTHOR), Lilleker, J.6,7 (AUTHOR), McConville, J.8 (AUTHOR), Merrison, A.9 (AUTHOR), Parton, M.10 (AUTHOR), Lofra, R. Muni1 (AUTHOR), Thorman, P.11 (AUTHOR), Scoto, M.2 (AUTHOR), Bettolo, C. Marini1 (AUTHOR) |
| Source: | Neuromuscular Disorders. 2025 Supplement, Vol. 53, pN.PAG-N.PAG. 1p. |
| Database: | Academic Search Ultimate |
| ISSN: | 09608966 |
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| DOI: | 10.1016/j.nmd.2025.105988 |
