ReviewJournal of medicine and life
Spinal muscular atrophy disease: a literature review for therapeutic strategies.
Review in Journal of medicine and life. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers, 2 of them syntheses that pooled it.
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, 2 syntheses or guidelines pooled it.
- Drug treatment for spinal muscular atrophy types II and III.The Cochrane database of systematic reviews · 2020Pooled it
- Drug treatment for spinal muscular atrophy type I.The Cochrane database of systematic reviews · 2019Pooled it
- Sorting differentiated mammalian cells using deterministic lateral displacement microfluidic devices.Analytical sciences : the international journal of the Japan Society for Analytical Chemistry · 2024Article
- Clinical applications of naturally derived biopolymer-based scaffolds for regenerative medicine.Annals of biomedical engineering · 2015Review
- Skeletal muscle tissue engineering: methods to form skeletal myotubes and their applications.Tissue engineering. Part B, Reviews · 2014Review
- Perceptions of equine-assisted activities and therapies by parents and children with spinal muscular atrophy.Pediatric physical therapy : the official publication of the Section on Pediatrics of the American Physical Therapy Association · 2014Article
- In vitro Differentiation of Functional Human Skeletal Myotubes in a Defined System.Biomaterials science · 2014Article
- Prenatal diagnosis of spinal muscular atrophy: clinical experience and molecular genetics of SMN gene analysis in 36 cases.Journal of prenatal medicine · 2013Article
- Genetic correction of human induced pluripotent stem cells from patients with spinal muscular atrophy.Science translational medicine · 2012Article
- Comparative effects between bone marrow and mesenchymal stem cell transplantation in GDNF expression and motor function recovery in a motorneuron degenerative mouse model.Stem cell reviews and reports · 2012Article
- Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy.Human molecular genetics · 2012Article
- Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.Transgenic research · 2011Article
- Neuromuscular junction formation between human stem cell-derived motoneurons and human skeletal muscle in a defined system.Biomaterials · 2011Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Currently, there is no cure for the treatment of spinal muscular atrophy (SMA). Based on the available clinical and molecular findings, different therapeutic strategies were tested in vitro and in vivo and clinical trials are ongoing. The main therapeutic direction is focused on the enhancement of SMN expression by increasing the full-length (fl) SMN2 transcript levels, preventing the SMN exon 7 from skipping or from protein stabilizing. In addition, the action of neurotrophic, neuroprotective or anabolic agents is tested and stem cell and gene therapy approaches are in a promising development.
Indexed as
Identifiers
20302191PMC3019038What 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.