Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec

Autor: Mercuri, Eugenio, Kirschner, Janbernd, De Vivo, Darryl C., Saito, Kayoko, Raju, Dheeraj, Benguerba, Kamal, Dabbous, Omar, Mumneh, Nayla, Reyna, Sandra P., Faulkner, Eric, Tizzano, Eduardo F., Quijano-Roy, Susana, Servais, Laurent, Parsons, Julie A., Aharoni, Sharon, Lakhotia, Arpita, Finkel, Richard S.
Zdroj: In European Journal of Paediatric Neurology November 2024 53:18-24
Databáze: ScienceDirect
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