ReviewTherapeutic advances in neurological disorders2018
Advances in spinal muscular atrophy therapeutics.
Review 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 42 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
42 citing papers in PubMed.
- Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids.Nature communications · 2025Article
- Real-world evidence of Nusinersen treatment for patients with spinal muscular atrophy in the Kingdom of Saudi Arabia: Initial insights from the Saudi national spinal muscular atrophy program.Saudi medical journal · 2025Article
- Comorbidities in spinal muscular atrophy and their impact on the course of the underlying disease: a real-life observational study.BMC musculoskeletal disorders · 2025Observational
- Lafora Disease: A Case Report and Evolving Treatment Advancements.Brain sciences · 2023Article
- Multi-omics profiling of CSF from spinal muscular atrophy type 3 patients after nusinersen treatment: a 2-year follow-up multicenter retrospective study.Cellular and molecular life sciences : CMLS · 2023Article
- Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources.Advances in therapy · 2023Article
- Developing antisense oligonucleotides for aMolecular therapy. Nucleic acids · 2022Article
- MicroRNA expression in animal models of amyotrophic lateral sclerosis and potential therapeutic approaches.Neural regeneration research · 2022Review
- Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Article
- Nanocarriers for Delivery of Oligonucleotides to the CNS.International journal of molecular sciences · 2022Review
- Perspectives on hiPSC-Derived Muscle Cells as Drug Discovery Models for Muscular Dystrophies.International journal of molecular sciences · 2021Review
- Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review.International journal of molecular sciences · 2021Review
- The informed road map to prevention of Alzheimer Disease: A call to arms.Molecular neurodegeneration · 2021Review
- Spinal Muscular Atrophy: Mutations, Testing, and Clinical Relevance.The application of clinical genetics · 2021Review
- 2020 Update to Spinal Muscular Atrophy Management in Saudi Arabia.Frontiers in pediatrics · 2021Article
- Evaluation of a Case Management to Support Families With Children Diagnosed With Spinal Muscular Atrophy-Protocol of a Controlled Mixed-Methods Study.Frontiers in pediatrics · 2021Article
- Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB.Pharmaceutics · 2020Review
- Genetic modifiers and phenotypic variability in neuromuscular disorders.Journal of applied genetics · 2020Review
- A National Spinal Muscular Atrophy Registry for Real-World Evidence.The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques · 2020Observational
- miR-206 Reduces the Severity of Motor Neuron Degeneration in the Facial Nuclei of the Brainstem in a Mouse Model of SMA.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
Corrections and comments
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Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Spinal muscular atrophy (SMA) is a progressive, recessively inherited neuromuscular disease, characterized by the degeneration of lower motor neurons in the spinal cord and brainstem, which leads to weakness and muscle atrophy. SMA currently represents the most common genetic cause of infant death. SMA is caused by the lack of survival motor neuron (SMN) protein due to mutations, which are often deletions, in the
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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.