RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy - rationale and study design

RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy - rationale and study design

<p><strong>Background:</strong> Dramatic improvements in spinal muscular atrophy (SMA) treatment have changed the prognosis for patients with this disease, leading to important new questions. Gathering representative, real-world data about the long-term efficacy and safety of emerg...

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Main Authors: 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
Format: Journal article
Sprog:English
Udgivet: IOS Press 2020

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