ReviewMuscle & nerve2011
Therapeutics development in myotonic dystrophy type 1.
Review in Muscle & nerve, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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
24 citing papers in PubMed, 49 citations in OpenAlex.
- Disruptions of cell signaling pathways in myotonic dystrophy type 1 skeletal muscle, their pathogenic impact, and potential for combinatorial therapeutics.The Journal of biological chemistry · 2026Review
- miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1.Molecular therapy. Methods & clinical development · 2021Article
- Resveratrol corrects aberrant splicing ofNeural regeneration research · 2020Article
- Responsiveness of outcome measures in myotonic dystrophy type 1.Annals of clinical and translational neurology · 2020Article
- miR-7 Restores Phenotypes in Myotonic Dystrophy Muscle Cells by Repressing Hyperactivated Autophagy.Molecular therapy. Nucleic acids · 2020Article
- Increased Muscleblind levels by chloroquine treatment improve myotonic dystrophy type 1 phenotypes in in vitro and in vivo models.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- Bioactive Aliphatic Polycarbonates Carrying Guanidinium Functions: An Innovative Approach for Myotonic Dystrophy Type 1 Therapy.ACS omega · 2019Article
- miR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models.Nature communications · 2018Article
- Development of pharmacophore models for small molecules targeting RNA: Application to the RNA repeat expansion in myotonic dystrophy type 1.Bioorganic & medicinal chemistry letters · 2016Article
- Functional KCa1.1 channels are crucial for regulating the proliferation, migration and differentiation of human primary skeletal myoblasts.Cell death & disease · 2016Article
- A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy.ChemMedChem · 2016Article
- Tibialis anterior muscle needle biopsy and sensitive biomolecular methods: a useful tool in myotonic dystrophy type 1.European journal of histochemistry : EJH · 2015Article
- Myotonic dystrophy type 1 associated with white matter hyperintense lesions: clinic, imaging, and genetic analysis.Chinese medical journal · 2015Article
- Natural history of skeletal muscle involvement in myotonic dystrophy type 1: a retrospective study in 204 cases.Journal of neurology · 2015Article
- Myotonic Dystrophy Health Index: initial evaluation of a disease-specific outcome measure.Muscle & nerve · 2014Article
- Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1.Journal of medicinal chemistry · 2013Article
- Diagnostic odyssey of patients with myotonic dystrophy.Journal of neurology · 2013Article
- Chronic muscle stimulation improves muscle function and reverts the abnormal surface EMG pattern in myotonic dystrophy: a pilot study.Journal of neuroengineering and rehabilitation · 2013Article
- Antisense therapy in neurology.Journal of personalized medicine · 2013Review
- Single-molecule study of the CUG repeat-MBNL1 interaction and its inhibition by small molecules.Nucleic acids research · 2013Article
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
Myotonic dystrophy (DM1), the most common adult muscular dystrophy, is a multisystem, autosomal dominant genetic disorder caused by an expanded CTG repeat that leads to nuclear retention of a mutant RNA and subsequent RNA toxicity. Significant insights into the molecular mechanisms of RNA toxicity have led to the previously unforeseen possibility that treating DM1 is a viable prospect. In this review, we briefly present the clinical picture in DM1, and describe how the research in understanding the pathogenesis of RNA toxicity in DM1 has led to targeted approaches to therapeutic development at various steps in the pathogenesis of the disease. We discuss the promise and current limitations of each with an emphasis on RNA-based therapeutics and small molecules. We conclude with a discussion of the unmet need for clinical tools and outcome measures that are essential prerequisites to proceed in evaluating these potential therapies in clinical trials.
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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.