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Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys

Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children < 2 years. For later-onset patients, intrathecal onasemnogene abeparvovec may be advantageous over intravenous administration. Recently, microscopic dorsal root ganglion (DRG) changes were ob...

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Detalles Bibliográficos
Autores principales: Tukov, Francis Fonyuy, Mansfield, Keith, Milton, Mark, Meseck, Emily, Penraat, Kelley, Chand, Deepa, Hartmann, Andreas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9347375/
https://www.ncbi.nlm.nih.gov/pubmed/35331006
http://dx.doi.org/10.1089/hum.2021.255