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Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies by Fischer R, Furlong P, Kennedy A, Maynard K, Penrod M, Miller D, Laverty CG, Lowes LP, Kuntz NL, Shieh PB, Kondejewski J, Neumann PJ, Shafrin J, Willke RJ
Published 2024-08-01
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RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy - rationale and study design by Finkel, RS, Day, JW, De Vivo, DC, Kirschner, J, Mercuri, E, Muntoni, F, Shieh, PB, Tizzano, E, Desguerre, I, Quijano-Roy, S, Saito, K, Droege, M, Dabbous, O, Khan, F, Renault, L, Anderson, FA, Servais, L
Published 2020Journal article -
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Nusinersen versus sham control in later-onset spinal muscular atrophy by Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS
Published 2018Journal article