ReviewCurrent rheumatology reports2011
The muscular dystrophies: distinct pathogenic mechanisms invite novel therapeutic approaches.
Review in Current rheumatology reports, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 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
5 citing papers in PubMed.
- Microalgae Produce Antioxidant Molecules with Potential Preventive Effects on Mitochondrial Functions and Skeletal Muscular Oxidative Stress.Antioxidants (Basel, Switzerland) · 2023Review
- Biological scaffold-mediated delivery of myostatin inhibitor promotes a regenerative immune response in an animal model of Duchenne muscular dystrophy.The Journal of biological chemistry · 2018Article
- Dystrophin Cardiomyopathies: Clinical Management, Molecular Pathogenesis and Evolution towards Precision Medicine.Journal of clinical medicine · 2018Review
- Making gene editing a therapeutic reality.F1000Research · 2018Review
- Novel tyrosine phosphorylation sites in rat skeletal muscle revealed by phosphopeptide enrichment and HPLC-ESI-MS/MS.Journal of proteomics · 2012Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Over the past decade, the enigmatic pathogenic mechanisms of the most common forms of muscular dystrophy have been defined. In this report, the molecular defects for each of these disorders are fully described, demonstrating the potential for therapeutic intervention. In facioscapulohumeral muscular dystrophy, recent findings implicate a stabilized DUX4 transcript within the contracted D4Z4 repeats, opening the door for an RNA interference treatment strategy. In the myotonic dystrophies (dystrophica myotonia [DM]), two variants of the disease (DM1 and DM2) are caused by unrelated genes yet manifest overlapping phenotypes. The common mechanism is a splicing disorder related to RNA toxicity. Duchenne muscular dystrophy is the most common childhood form of muscular dystrophy. In many ways, the molecular gene defects are the most traditional. Gene repair strategies have advanced to the level of clinical testing, and we hope they will provide relief for this most devastating form of muscular dystrophy.
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Registered trials
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