ArticleTherapeutic advances in neurological disorders2018
Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy.
Article in Therapeutic advances in neurological disorders, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers, 1 of them a synthesis that pooled it.
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The trial behind it
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Who cites it
34 citing papers in PubMed, 1 synthesis or guideline pooled it, 87 citations in OpenAlex.
- A Systematic Review of Procedural Complications from Transforaminal Lumbar Puncture for Intrathecal Nusinersen Administration in Patients with Spinal Muscular Atrophy.AJNR. American journal of neuroradiology · 2021Pooled it
- Advanced Drug Delivery in Pediatric Neurology: Developmental Barriers, Nanocarrier Strategies, and Translational Challenges.International journal of molecular sciences · 2026Review
- Beyond Coding Variants: RNA-Level Mechanisms in Human Disease and Precision Therapeutics.Genes · 2026Review
- Defining When Nusinersen Starts to Work: Time to Clinical Benefit in Patients with SMA Types 1-3 from a Real-World Cohort in China.Diagnostics (Basel, Switzerland) · 2026Article
- 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
- Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam.Journal of neurology · 2026Article
- Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience.Frontiers in pediatrics · 2026Article
- Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China.BMC pediatrics · 2025Article
- Nusinersen Improves Motor Function in Type 2 and 3 Spinal Muscular Atrophy Patients across Time.Biomedicines · 2024Article
- Improvement in functional motor scores in patients with non-ambulatory spinal muscle atrophy during Nusinersen treatment in South Korea: a single center study.BMC neurology · 2024Article
- Risdiplam improves subjective swallowing quality in non-ambulatory adult patients with 5q-spinal muscular atrophy despite advanced motor impairment.Journal of neurology · 2024Article
- Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety.Journal of personalized medicine · 2024Article
- Nusinersen for spinal muscular atrophy types II and III: a retrospective single-center study in South Korea.World journal of pediatrics : WJP · 2023Article
- Improvement of muscle strength in specific muscular regions in nusinersen-treated adult patients with 5q-spinal muscular atrophy.Scientific reports · 2023Article
- Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.Neurology and therapy · 2023Article
- [Gene-based treatment in spinal muscular atrophy].Der Nervenarzt · 2022Review
- Nusinersen Treatment in Adults With Spinal Muscular Atrophy.Neurology. Clinical practice · 2021Article
- Article
Corrections and comments
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Authors and funding
11 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundNusinersen is an intrathecally administered antisense oligonucleotide (ASO) and the first approved drug for the treatment of spinal muscular atrophy (SMA). However, progressive neuromyopathic scoliosis and the presence of spondylodesis can impede lumbar punctures in SMA patients. Our aim was to assess the feasibility and safety of the treatment in adults with SMA.
methodsFor the intrathecal administration of nusinersen, we performed conventional, fluoroscopy-assisted and computer tomography (CT)-guided lumbar punctures in adult patients with type 2 and type 3 SMA. We documented any reported adverse events and performed blood tests.
resultsWe treated a total of 28 adult SMA patients (9 patients with SMA type 2 and 19 patients with SMA type 3) aged between 18-61
conclusionsOur data demonstrate the feasibility and tolerability of intrathecal treatment with nusinersen in adults with SMA type 2 and type 3. However, treatment can be medically and logistically challenging, particularly in patients with SMA type 2 and in patients with spondylodesis.
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