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

Tizzano, E. F., Quijano-Roy, S., Servais, L., Parsons, J. A., Aharoni, S., Lakhotia, A., Finkel, R. S., Mercuri, E., Kirschner, J., De Vivo, D. C., 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, 53, 18–24. https://doi.org/10.1016/j.ejpn.2024.08.006
Authors:
Eduardo F. Tizzano
Susana Quijano-Roy
Laurent Servais
Julie Parsons
Sharon Aharoni
Arpita Lakhotia
Richard S. Finkel
Eugenio Mercuri
Janbernd Kirschner
Darryl C. De Vivo
Kayoko Saito
Dheeraj Raju
Kamal Benguerba
Omar Dabbous
N. Mumneh
Sandra P. Reyna
Eric Faulkner
Affiliated Authors:
Darryl C. De Vivo
Columbia Affiliation:
Subjects:
Survival of Motor Neuron 2 Protein (MeSH)
Registries (MeSH)
Muscular Atrophy, Spinal (MeSH)
Gene Therapy for Spinal Muscular Atrophy (OpenAlex Topic)
Etiology and Management of Abdominal Wall Defects (OpenAlex Topic)
Impact of Parenting Stress on Caregivers and Families (OpenAlex Topic)
Author Keywords:
four smn2 gene copies
onasemnogene abeparvovec
real-world data
restore registry
spinal muscular atrophy
survival motor neuron 2 gene
elink
Publication Type:
Article
Unique ID:
10.1016/j.ejpn.2024.08.006
PMID:
39260228
DOI:
10.1016/j.ejpn.2024.08.006
Journal:
European Journal of Paediatric Neurology
Publication Date:
Data Source:
OpenAlex
Source Link:
View OpenAlex Record
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Record Created: