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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Veröffentlicht in:	Journal of pediatric orthopaedics 2018-11, Vol.38 (10), p.e610-e617
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Catheterization - instrumentation Child Female Humans Injections, Spinal - methods Male Muscular Atrophy, Spinal - drug therapy Neuromuscular Oligonucleotides - administration & dosage Outpatients Pain Management Quality of Life Spinal Fusion - adverse effects
Online-Zugang:	Volltext
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