ReviewClinical drug investigation2017
Nusinersen: The First Option Beyond Supportive Care for Spinal Muscular Atrophy.
Review in Clinical drug investigation, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
13 citing papers in PubMed, 25 citations in OpenAlex.
- Risdiplam therapy in adult patients with Spinal muscular Atrophy (SMA). A 24-month-real-world experience at a single muscular centre.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2026Article
- RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System.Biomolecules & therapeutics · 2025Review
- Inhibition of Rho-Associated Kinases ROCK1 and ROCK2 as a Therapeutic Strategy to Reactivate the RepressedThe Journal of neuroscience : the official journal of the Society for Neuroscience · 2025Article
- The Impact of Nusinersen Treatment on Respiratory Function in Patients with Spinal Muscular Atrophy: A Systematic Review.Journal of clinical medicine · 2024Review
- Antisense Oligonucleotide Technologies to Combat Obesity and Fatty Liver Disease.Frontiers in physiology · 2022Review
- A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome.Nature medicine · 2021Article
- Targeting for Success: Demonstrating Proof-of-Concept with Mechanistic Early Phase Clinical Pharmacology Studies for Disease-Modification in Neurodegenerative Disorders.International journal of molecular sciences · 2021Review
- Cervical rotation, chest deformity and pelvic obliquity in patients with spinal muscular atrophy.BMC musculoskeletal disorders · 2020Article
- Silencing ofPhysiological genomics · 2020Article
- Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders.Frontiers in genetics · 2019Review
- Alternative mRNA Splicing in the Pathogenesis of Obesity.International journal of molecular sciences · 2018Review
- Pharmacological c-Jun NHFrontiers in molecular neuroscience · 2018Article
- Nusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAGReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterized by degeneration of spinal motor neurons and poses significant adverse outcome in affected population. Survival motor neuron 1 (SMN1) protein encoded by SMN1 gene located on 5q
Indexed as
Identifiers
What OpenQuestion holds
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.