Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy

BACKGROUND: Many patients with spinal muscular atrophy (SMA) who might benefit from intrathecal antisense oligonucleotide (nusinersen) therapy have scoliosis or spinal fusion that precludes safe drug delivery. To circumvent spinal pathology, we designed a novel subcutaneous intrathecal catheter (SIC...

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Detalles Bibliográficos
Autores principales: Strauss, Kevin A., Carson, Vincent J., Brigatti, Karlla W., Young, Millie, Robinson, Donna L., Hendrickson, Christine, Fox, Michael D., Reed, Robert M., Puffenberger, Erik G., Mackenzie, William, Miller, Freeman
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2018
Materias:
Neuromuscular
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6211782/
https://www.ncbi.nlm.nih.gov/pubmed/30134351
http://dx.doi.org/10.1097/BPO.0000000000001247

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6211782/
https://www.ncbi.nlm.nih.gov/pubmed/30134351
http://dx.doi.org/10.1097/BPO.0000000000001247

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