ReviewCurrent neurology and neuroscience reports2017
Treatment Advances in Spinal Muscular Atrophy.
Review in Current neurology and neuroscience reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed, 43 citations in OpenAlex.
- When barriers fail: the role of endothelial dysfunction in rare pediatric neuromuscular diseases.Frontiers in medicine · 2026Review
- Analysis of spinal muscular atrophy patients from the spinal muscular atrophy and muscular dystrophy registry of Pakistan.Journal of neuromuscular diseases · 2025Article
- Sociodemographic and clinical characteristics and access to health care in patients with spinal muscular atrophy in Argentina.Frontiers in neurology · 2023Review
- A comparative analysis of body composition assessment by BIA and DXA in children with type II and III spinal muscular atrophy.Frontiers in neurology · 2022Article
- Using gene panels in the diagnosis of neuromuscular disorders: A mini-review.Frontiers in neurology · 2022Review
- Axonal mRNA localization and local translation in neurodegenerative disease.Neural regeneration research · 2021Review
- Engineering Crystal Packing in RNA-Protein Complexes II: A Historical Perspective from the Structural Studies of the Spliceosome.Crystals · 2021Article
- Molecular Biomarkers for Spinal Muscular Atrophy: A Systematic Review.Neurology. Clinical practice · 2021Review
- Axonal Transport and Local Translation of mRNA in Neurodegenerative Diseases.Frontiers in molecular neuroscience · 2021Review
- SMA Identified: Clinical and Molecular Findings From a Sponsored Testing Program for Spinal Muscular Atrophy in More Than 2,000 Individuals.Frontiers in neurology · 2021Article
- The Antisense Oligonucleotide Nusinersen for Treatment of Spinal Muscular Atrophy.Orthopedic reviews · 2021Review
- Economic burden of spinal muscular atrophy: an analysis of claims data.Journal of market access & health policy · 2020Article
- Article
- Inhibition of myostatin prevents microgravity-induced loss of skeletal muscle mass and strength.PloS one · 2020Article
- [Treatment evaluation in patients with 5q-associated spinal muscular atrophy : Real-world experience].Der Nervenarzt · 2019Review
- Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy.Frontiers in neurology · 2019Review
- RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis.Biomedicines · 2018Review
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 2 institutions in 1 country.
Funding
Abstract
purpose of reviewSpinal muscular atrophy (SMA) is a genetic disorder of motor neurons in the anterior horns of the spinal cord and brainstem that results in muscle atrophy and weakness. SMA is an autosomal recessive disease linked to deletions of the SMN1 gene on chromosome 5q. Humans have a duplicate gene (SMN2) whose product can mitigate disease severity, leading to the variability in severity and age of onset of disease, and is therefore a target for drug development. RECENT
findingsAdvances in preclinical and clinical trials have paved the way for novel therapeutic options for SMA patients, including many currently in clinical trials. In 2016, the first treatment for SMA has been approved in the USA, an antisense oligonucleotide that increases full-length protein product derived from SMN2. The approval of a first treatment for SMA and the rapid advances in clinical trials provide the prospect for multiple approaches to disease modification. There are several other promising therapeutics in different stages of development, based on approaches such as neuroprotection, or gene therapy.
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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.