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 | Semantic Scholar Skip to search formSkip to main content> Semantic Scholar's Logo Search Sign InCreate Free Account You are currently offline. Some features of the site may not work correctly. DOI:10.1097/BPO.0000000000001247 Corpus ID: 52069305Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy @article{Strauss2018PreliminarySA, title={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}, author={K. Strauss and V. Carson and Karlla W. Brigatti and M. Young and D. Robinson and Christine L Hendrickson and M. Fox and R. Reed and E. Puffenberger and William Mackenzie and F. Miller}, journal={Journal of Pediatric Orthopedics}, year={2018}, volume={38}, pages={e610 - e617} } K. Strauss, V. Carson, +8 authors F. Miller Published 2018 Medicine Journal of Pediatric Orthopedics 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) system by connecting an intrathecal catheter to an implantable infusion port. Methods: Device safety and tolerability were tested in 10 SMA patients (age, 5.4 to 30.5 y; 80% with 3 copies of SMN2… Expand View on Wolters Kluwer europepmc.org Save to Library Create Alert Cite Launch Research Feed Share This Paper 16 CitationsBackground Citations 3 Methods Citations 1 View All Topics from this paper nusinersen Glucose Juvenile Spinal Muscular Atrophy Specimen Atrophic Muscle Cognition Disorders Standard Industrial Classification antisense therapy Insurance Carriers Drug Delivery Systems Implants Blood Cells Scoliosis, unspecified Cerebrospinal Fluid Large Implantable venous access port Leukocytes Paper Mentions News Article SMA, nuovo sistema sicuro di somministrazione intratecale per nusi Pharmastar Italy 30 September 2018 News Article New nusinersen drug delivery method identified for spinal muscular atrophy patients EurekAlert! 24 August 2018 16 Citations Citation Type Citation Type All Types Cites Results Cites Methods Cites Background Has PDF Publication Type Author More Filters More Filters Filters Sort by Relevance Sort by Most Influenced Papers Sort by Citation Count Sort by Recency Multicenter Experience with Nusinersen Application via an Intrathecal Port and Catheter System in Spinal Muscular Atrophy. M. Flotats-Bastardas, A. Hahn, +4 authors C. Koehler Medicine Neuropediatrics 2020 Save Alert Research Feed Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1. Astrid Pechmann, T. Langer, S. Wider, J. Kirschner Medicine European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society 2018 17 Save Alert Research Feed Transforaminal intrathecal delivery of nusinersen for older children and adults with spinal muscular atrophy and complex spinal anatomy: an analysis of 200 consecutive injections J. Weaver, D. Hallam, +10 authors Eric J Monroe Medicine Journal of NeuroInterventional Surgery 2020 1 Save Alert Research Feed Intrathecal Administration of Nusinersen in Pediatric SMA Patients with and without Spine Deformities: Experiences and Challenges over 3 Years in a Single Center. J. Johannsen, Deike Weiss, F. Schlenker, M. Groth, J. Denecke Medicine Neuropediatrics 2020 Save Alert Research Feed Clinical Implication of Dosimetry of Computed Tomography- and Fluoroscopy-Guided Intrathecal Therapy With Nusinersen in Adult Patients With Spinal Muscular Atrophy K. Kizina, B. Stolte, +8 authors T. Hagenacker Medicine Front. Neurol. 2019 15 View 1 excerpt, cites background Save Alert Research Feed Nusinersen Administration Via an Intrathecal Port in a 16-Year-Old Spinal Muscular Atrophy Patient with Profound Scoliosis M. Flotats-Bastardas, S. Linsler, M. Zemlin, S. Meyer Medicine Pediatric Neurosurgery 2019 3 Save Alert Research Feed New treatments in spinal muscular atrophy: an overview of currently available data S. Ramdas, L. Servais Medicine Expert opinion on pharmacotherapy 2020 23 View 1 excerpt, cites background Save Alert Research Feed Children With Spinal Muscular Atrophy With Prior Growth-Friendly Spinal Implants Have Better Results After Definite Spinal Fusion in Comparison to Untreated Patients. A. Hell, L. Braunschweig, +4 authors H. M. Lorenz Medicine Neurosurgery 2020 2 Save Alert Research Feed Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment A. Monnette, Er Chen, +4 authors Lizheng Shi Medicine Orphanet Journal of Rare Diseases 2021 View 1 excerpt, cites background Save Alert Research Feed Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care D. Schorling, Astrid Pechmann, J. Kirschner Medicine Journal of neuromuscular diseases 2019 16 View 1 excerpt Save Alert Research Feed ... 1 2 ... References SHOWING 1-10 OF 38 REFERENCES SORT BYRelevance Most Influenced Papers Recency Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1. Astrid Pechmann, T. Langer, S. Wider, J. Kirschner Medicine European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society 2018 17 Save Alert Research Feed Clinical Implication of Dosimetry of Computed Tomography- and Fluoroscopy-Guided Intrathecal Therapy With Nusinersen in Adult Patients With Spinal Muscular Atrophy K. Kizina, B. Stolte, +8 authors T. Hagenacker Medicine Front. Neurol. 2019 15 Save Alert Research Feed Transforaminal intrathecal delivery of nusinersen using cone-beam computed tomography for children with spinal muscular atrophy and extensive surgical instrumentation: early results of technical success and safety J. Weaver, N. Natarajan, +4 authors E. Monroe Medicine Pediatric Radiology 2017 30 Save Alert Research Feed Intrathecal Injections in Children With Spinal Muscular Atrophy Manon Haché, K. Swoboda, +4 authors K. 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K. Benjamin Medicine Journal of neurosurgery 2007 22 Save Alert Research Feed Reliability of telephone administration of the PedsQL™ Generic Quality of Life Inventory™ and Neuromuscular Module™ in spinal muscular atrophy (SMA) S. Dunaway, J. Montes, M. Montgomery, V. Battista, P. Kaufmann Medicine Neuromuscular Disorders 2010 20 PDF Save Alert Research Feed ... 1 2 3 4 ... Related Papers Abstract Topics Paper Mentions 16 Citations 38 References Related Papers Stay Connected With Semantic Scholar Sign Up About Semantic Scholar Semantic Scholar is a free, AI-powered research tool for scientific literature, based at the Allen Institute for AI. 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