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

Eduardo F. Tizzano, Susana Quijano-Roy, Laurent Servais, Julie A. Parsons, Sharon Aharoni, Arpita Lakhotia, Richard S. Finkel, Eugenio Mercuri, Janbernd Kirschner, Darryl C. De Vivo, Kayoko Saito, Dheeraj Raju, Kamal Benguerba, Omar Dabbous, Nayla Mumneh, Sandra P. Reyna, Eric Faulkner

Open source

DOI: 10.1016/j.ejpn.2024.08.006
Published: 2024-11
Container: European Journal of Paediatric Neurology
Publisher: Elsevier BV
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/j.ejpn.2024.08.006,
  title = {Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec},
  author = {Eduardo F. Tizzano and Susana Quijano-Roy and Laurent Servais and Julie A. Parsons and Sharon Aharoni and Arpita Lakhotia and Richard S. Finkel and Eugenio Mercuri and Janbernd Kirschner and Darryl C. De Vivo and Kayoko Saito and Dheeraj Raju and Kamal Benguerba and Omar Dabbous and Nayla Mumneh and Sandra P. Reyna and Eric Faulkner},
  year = {2024},
  journal = {European Journal of Paediatric Neurology},
  doi = {10.1016/j.ejpn.2024.08.006},
  url = {https://doi.org/10.1016/j.ejpn.2024.08.006}
}

RIS

TY  - JOUR
TI  - Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec
AU  - Eduardo F. Tizzano
AU  - Susana Quijano-Roy
AU  - Laurent Servais
AU  - Julie A. Parsons
AU  - Sharon Aharoni
AU  - Arpita Lakhotia
AU  - Richard S. Finkel
AU  - Eugenio Mercuri
AU  - Janbernd Kirschner
AU  - Darryl C. De Vivo
AU  - Kayoko Saito
AU  - Dheeraj Raju
AU  - Kamal Benguerba
AU  - Omar Dabbous
AU  - Nayla Mumneh
AU  - Sandra P. Reyna
AU  - Eric Faulkner
PY  - 2024
JO  - European Journal of Paediatric Neurology
DO  - 10.1016/j.ejpn.2024.08.006
UR  - https://doi.org/10.1016/j.ejpn.2024.08.006
ER  -

APA

Tizzano, E. F., Quijano-Roy, S., Servais, L., Parsons, J. A., Aharoni, S., Lakhotia, A., Finkel, R. S., Mercuri, E., Kirschner, J., Vivo, D. C. D., Saito, K., Raju, D., Benguerba, K., Dabbous, O., Mumneh, N., Reyna, S. P., & Faulkner, E. (2024). Outcomes for patients in the RESTORE registry with spinal muscular atrophy and four or more SMN2 gene copies treated with onasemnogene abeparvovec. European Journal of Paediatric Neurology. https://doi.org/10.1016/j.ejpn.2024.08.006

Source records

crossref · retrieved 2026-09-26T10:06:07.757Z