ArticleTherapeutic advances in neurological disorders2010
Therapeutic developments in spinal muscular atrophy.
Article in Therapeutic advances in neurological disorders, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 22 citations in OpenAlex.
- The Relationship between Body Composition, Fatty Acid Metabolism and Diet in Spinal Muscular Atrophy.Brain sciences · 2021Review
- High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids' Inpatient Database (KID).PharmacoEconomics - open · 2019Article
- Overexpression of SMN2 Gene in Motoneuron-Like Cells Differentiated from Adipose-Derived Mesenchymal Stem Cells by Ponasterone A.Journal of molecular neuroscience : MN · 2019Article
- CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency.Science (New York, N.Y.) · 2019Article
- A New Method for Measuring Bell-Shaped Chest Induced by Impaired Ribcage Muscles in Spinal Muscular Atrophy Children.Frontiers in neurology · 2018Article
- Severe burn increased skeletal muscle loss in mdx mutant mice.The Journal of surgical research · 2016Article
- Quantitative muscle ultrasound measures rapid declines over time in children with SMA type 1.Journal of the neurological sciences · 2015Article
- Expression of Muscle-Specific MiRNA 206 in the Progression of Disease in a Murine SMA Model.PloS one · 2015Article
- 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
- Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds.EMBO molecular medicine · 2013Article
- Clinical and molecular cross-sectional study of a cohort of adult type III spinal muscular atrophy patients: clues from a biomarker study.European journal of human genetics : EJHG · 2013Article
- Identification of a Maleimide-Based Glycogen Synthase Kinase-3 (GSK-3) Inhibitor, BIP-135, that Prolongs the Median Survival Time of Δ7 SMA KO Mouse Model of Spinal Muscular Atrophy.ACS chemical neuroscience · 2012Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Spinal muscular atrophy (SMA), a potentially devastating disease marked by progressive weakness and muscle atrophy resulting from the dysfunction and loss of motor neurons of the spinal cord, has emerged in recent years as an attractive target for therapeutic intervention. Caused by a homozygous mutation to the Survival of Motor Neurons 1 (SMN1) gene on chromosome 5q, the severity of the clinical phenotype in SMA is modulated by the function of a related protein, Survival of Motor Neurons 2 (SMN2). SMN2 predominantly produces an unstable SMN transcript lacking exon 7; only about 10% of the transcription product produces a full-length, functional SMN protein. Several therapeutic strategies have targeted this gene with the goal of producing increased full-length SMN transcript, thereby modifying the underlying mechanism. Drugs that have increased SMN2 function, in vitro, are now explored for potential therapeutic benefit in this disease. Alternative approaches, including neuroprotective, muscle anabolic, gene and cell replacement strategies, also hold promise. The recent advances in preclinical research and the development of a wider range of animal models for SMA continue to provide cautious optimism that effective treatments for SMA will eventually emerge.
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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.