ReviewFuture medicinal chemistry2012
SMN-inducing compounds for the treatment of spinal muscular atrophy.
Review in Future medicinal chemistry, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 34 citations in OpenAlex.
- Target-Based Discovery of Drug Combinations for Spinal Muscular Atrophy.Advances in experimental medicine and biology · 2026Article
- Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review.International journal of molecular sciences · 2021Review
- The Relationship between Body Composition, Fatty Acid Metabolism and Diet in Spinal Muscular Atrophy.Brain sciences · 2021Review
- Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases.International journal of molecular sciences · 2020Review
- Functional characterization of SMN evolution in mouse models of SMA.Scientific reports · 2019Article
- Advances in therapy for spinal muscular atrophy: promises and challenges.Nature reviews. Neurology · 2018Review
- Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.Drugs · 2018Review
- Article
- SAM68 is a physiological regulator of SMN2 splicing in spinal muscular atrophy.The Journal of cell biology · 2015Article
- Spinal muscular atrophy--recent therapeutic advances for an old challenge.Nature reviews. Neurology · 2015Review
- Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.Human molecular genetics · 2014Article
- Defining the therapeutic window in a severe animal model of spinal muscular atrophy.Human molecular genetics · 2014Article
- Assays for the identification and prioritization of drug candidates for spinal muscular atrophy.Assay and drug development technologies · 2014Review
- Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation.The Journal of clinical investigation · 2014Article
- Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?Journal of anatomy · 2014Review
- The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.Human molecular genetics · 2013Article
- Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.EMBO molecular medicine · 2013Article
- A cell system for phenotypic screening of modifiers of SMN2 gene expression and function.PloS one · 2013Article
- Article
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
Abstract
Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. A neurodegenerative disease, it is caused by loss of SMN1, although low, but essential, levels of SMN protein are produced by the nearly identical gene SMN2. While no effective treatment or therapy currently exists, a new wave of therapeutics has rapidly progressed from cell-based and preclinical animal models to the point where clinical trials have initiated for SMA-specific compounds. There are several reasons why SMA has moved relatively rapidly towards novel therapeutics, including: SMA is monogenic; the molecular understanding of SMN gene regulation has been building for nearly 20 years; and all SMA patients retain one or more copies of SMN2 that produces low levels of full-length, fully functional SMN protein. This review primarily focuses upon the biology behind the disease and examines SMN1- and SMN2-targeted therapeutics.
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.