ArticleJournal of neurology2019
Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients.
Article in Journal of neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 53 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
53 citing papers in PubMed, 116 citations in OpenAlex.
- Estimation of human brain fluid clearance using intrathecal imaging contrast agents.Fluids and barriers of the CNS · 2026Observational
- CT-guided Lumbar Puncture for Intrathecal Nusinersen Injection in Patients with Spinal Muscular Atrophy: Technical Effectiveness, Safety, and Radiation Dose.Clinical neuroradiology · 2026Article
- Risdiplam Impact in Treatment Naïve and Non-Naïve Pediatric and Adult Patients With Spinal Muscular Atrophy.European journal of neurology · 2026Article
- Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study.BMC neurology · 2026Observational
- The impact of human brain geometry on the transport of an intrathecal tracer.Fluids and barriers of the CNS · 2026Article
- Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study.Orphanet journal of rare diseases · 2025Observational
- Surgical Access for Intrathecal Therapy in Spinal Muscular Atrophy with Spinal Fusion: Long-Term Outcomes of Lumbar Laminectomy.Journal of clinical medicine · 2025Article
- Utilization of 3D printing modeling techniques in the simulation instruction of ultrasound-guided puncture procedures on scoliotic spines of spinal muscular atrophy.3D printing in medicine · 2025Article
- Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China.BMC pediatrics · 2025Article
- Considerations for repetitive intrathecal procedures in long-term nusinersen treatment for non-ambulatory spinal muscular atrophy.Scientific reports · 2025Article
- Review
- CDC's Laboratory Activities to Support Newborn Screening for Spinal Muscular Atrophy.International journal of neonatal screening · 2024Article
- Respiratory function in adult patients with spinal muscular atrophy treated with nusinersen - a monocenter observational study.Frontiers in neurology · 2024Article
- Development of a population pharmacokinetic model to characterize the pharmacokinetics of intrathecally administered tominersen in cerebrospinal fluid and plasma.CPT: pharmacometrics & systems pharmacology · 2023Article
- Nusinersen for spinal muscular atrophy types II and III: a retrospective single-center study in South Korea.World journal of pediatrics : WJP · 2023Article
- CNS Delivery of Nucleic Acid Therapeutics: Beyond the Blood-Brain Barrier and Towards Specific Cellular Targeting.Pharmaceutical research · 2023Review
- Antisense oligonucleotides: a novel Frontier in pharmacological strategy.Frontiers in pharmacology · 2023Review
- Role of circulating biomarkers in spinal muscular atrophy: insights from a new treatment era.Frontiers in neurology · 2023Review
- Non-invasive Imaging of Antisense Oligonucleotides in the Brain via In Vivo Click Chemistry.Molecular imaging and biology · 2022Article
- Population pharmacokinetic modeling of CSF to blood clearance: prospective tracer study of 161 patients under work-up for CSF disorders.Fluids and barriers of the CNS · 2022Article
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy is a genetic motor neuron disease that leads to progressive muscular atrophy and muscle weakness. In December 2016, the Food and Drug Administration, and in June 2017, the European Medicines Agency approved the antisense oligonucleotide nusinersen for treatment of spinal muscular atrophy. Nusinersen has to be repeatedly administered intrathecally. Due to the clinical features of SMA, the application of the ASO by lumbar puncture can be challenging in symptomatic patients considering the frequently observed scoliosis, previous spine fusion surgeries, joint contractures, and respiratory insufficiency. To evaluate safety and feasibility of the intrathecal treatment in adolescent and adult SMA type 2 and 3 patients, we analyzed 93 lumbar punctures, monitored number of lumbar puncture attempts, duration of the procedure, injection site, and needle length. Oxygen saturation during the intervention, medication for sedation and local anesthesia, adverse events related to lumbar punctures, and macroscopic analysis of CSF were recorded. Moreover, we analyzed the use of CT-scans for performing lumbar punctures and its associated radiation exposure. Performing lumbar puncture for the intrathecal administration of nusinersen in adolescent and adult patients with later-onset SMA is feasible and safe, even in patients with complex spinal anatomies and respiratory insufficiency. To guarantee the quality of the procedure, we recommend establishing an experienced interdisciplinary team consisting of neurologists and/or neuropediatricians, anesthesiologists, orthopedic surgeons, and/or neuroradiologists.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.