Journal of Neuromuscular Diseases
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Rodriguez-Torres, R., Kanner, C. H., Gay, E. L., Uher, D., Corbeil, T., Coratti, G., Dunaway Young, S., Rohwer, A., Muni Lofra, R., McDermott, M. P., De Vivo, D. C., Wall, M. M., Glynn, N. W., & Montes, J. (2025). Development of the SMA EFFORT: A new approach to characterize perceived physical fatigability in spinal muscular atrophy. Journal of Neuromuscular Diseases, 12(1). https://doi.org/10.1177/22143602241313326
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Wolfe, A., Stimpson, G., Ramsey, D., Coratti, G., Dunaway Young, S., Mayhew, A., Pane, M., Rohwer, A., Muni Lofra, R., Duong, T., O’Reilly, E., Milev, E., Civitello, M., Sansone, V. A., D’Amico, A., Bertini, E., Messina, S., Bruno, C., … Albamonte, E. (2024). Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3. Journal of Neuromuscular Diseases, 11(3), 665–677. https://doi.org/10.3233/jnd-230211
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Servais, L., Day, J. W., De Vivo, D. C., Kirschner, J., Mercuri, E., Muntoni, F., Proud, C. M., Shieh, P. B., Tizzano, E. F., Quijano-Roy, S., Desguerre, I., Saito, K., Faulkner, E., Benguerba, K. M., Raju, D., LaMarca, N., Sun, R., Anderson, F. A., & Finkel, R. S. (2024). Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry. Journal of Neuromuscular Diseases, 11(2), 425–442. https://doi.org/10.3233/jnd-230122
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Finkel, R. S., Day, J. W., Pascual Pascual, S. I., Ryan, M. M., Mercuri, E., De Vivo, D. C., Montes, J., Gurgel-Giannetti, J., Monine, M., Gambino, G., Makepeace, C., Foster, R., Berger, Z., & DEVOTE Study Group (2023). DEVOTE Study Exploring Higher Dose of Nusinersen in Spinal Muscular Atrophy: Study Design and Part A Results. Journal of neuromuscular diseases, 10(5), 813–823. https://doi.org/10.3233/JND-221667
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Uher, D., Yoon, L., Garber, C. E., & Montes, J. (2023). How is Physical Activity Measured in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy? Journal of Neuromuscular Diseases, 10(5), 897–914. https://doi.org/10.3233/jnd-230033
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Berardo, A., Domínguez-González, C., Engelstad, K., & Hirano, M. (2022). Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. Journal of Neuromuscular Diseases, 9(2), 225–235. https://doi.org/10.3233/jnd-210786
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