Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Servais, L., Day, J. W., De Vivo, D. C., Kirschner, J., Mercuri, E., Muntoni, F., Proud, C. M., Shieh, P. B., Tizzano, E. F., Quijano-Roy, S., Desguerre, I., Saito, K., Faulkner, E., Benguerba, K. M., Raju, D., LaMarca, N., Sun, R., Anderson, F. A., & Finkel, R. S. (2024). Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry. Journal of Neuromuscular Diseases, 11(2), 425–442. https://doi.org/10.3233/jnd-230122
Authors:
Laurent Servais
John W Day
Darryl C De Vivo
Janbernd Kirschner
Eugenio Mercuri
Francesco Muntoni
Crystal M Proud
Perry B Shieh
Eduardo F Tizzano
Susana Quijano-Roy
Isabelle Desguerre
Kayoko Saito
Eric Faulkner
Kamal M Benguerba
Dheeraj Raju
Nicole LaMarca
Rui Sun
Frederick A Anderson
Richard S Finkel
Affiliated Authors:
Darryl C De Vivo
Columbia Affiliation:
Subjects:
Gene Therapy for Spinal Muscular Atrophy (OpenAlex Topic)
Etiology and Management of Abdominal Wall Defects (OpenAlex Topic)
Spinal Muscular Atrophies of Childhood (MeSH)
Muscular Atrophy, Spinal (MeSH)
Biological Products (MeSH)
Recombinant Fusion Proteins (MeSH)
Author Keywords:
aav9 vector-based gene replacement therapy
long-term follow-up
motor neuron disease
newborn screening
onasemnogene abeparvovec
outcomes
rare disease
real-world evidence
restore registry
spinal muscular atrophy
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Publication Type:
Article
Unique ID:
10.3233/jnd-230122
PMID:
38250783
DOI:
10.3233/jnd-230122
Journal:
Journal of Neuromuscular Diseases
Publication Date:
Data Source:
PubMed
Source Link:
View PubMed Record
View OpenAlex Record
View Scopus Record
View Web of Science Record

Record Created: