ArticleCurrent pediatrics reports2014
Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.
Article in Current pediatrics reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed.
- Quantifying disease activity in fatty-infiltrated skeletal muscle by IDEAL-CPMG in Duchenne muscular dystrophy.Neuromuscular disorders : NMD · 2016Trial
- Dystrophin- and Utrophin-Based Therapeutic Approaches for Treatment of Duchenne Muscular Dystrophy: A Comparative Review.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- A New Functional Scale and Ambulatory Functional Classification of Duchenne Muscular Dystrophy: Scale Development and Preliminary Analyses of Reliability and Validity.Annals of rehabilitation medicine · 2018Article
- Whole-body clearing, staining and screening of calcium deposits in the mdx mouse model of Duchenne muscular dystrophy.Skeletal muscle · 2018Article
- Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.The Journal of biological chemistry · 2018Article
- An Evidence-Based, Community-Engaged Approach to Develop an Interactive Deliberation Tool for Pediatric Neuromuscular Trials.Journal of genetic counseling · 2018Article
- Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders.Clinical trials (London, England) · 2018Article
- Genome engineering: a new approach to gene therapy for neuromuscular disorders.Nature reviews. Neurology · 2017Review
- Recent developments in Duchenne muscular dystrophy: facts and numbers.Journal of cachexia, sarcopenia and muscle · 2017Article
- [A retrospective analysis of 6 children with Duchenne muscular dystrophy].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2017Article
- Current Translational Research and Murine Models For Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2016Review
- piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.Nucleic acids research · 2016Article
- Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy.Human gene therapy · 2015Review
- Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.EMBO molecular medicine · 2015Article
- Duchenne Muscular Dystrophy: From Diagnosis to Therapy.Molecules (Basel, Switzerland) · 2015Review
- Viral vector-mediated gene therapies.Current opinion in neurology · 2015Review
- Comparison of mutation profiles in the Duchenne muscular dystrophy gene among populations: implications for potential molecular therapies.International journal of molecular sciences · 2015Article
- A reporter mouse for optical imaging of inflammation in mdx muscles.Skeletal muscle · 2015Article
- The role of antisense oligonucleotide therapy in patients with familial hypercholesterolemia: risks, benefits, and management recommendations.Current atherosclerosis reports · 2015Review
- Skeletal muscle fibrosis in the mdx/utrn+/- mouse validates its suitability as a murine model of Duchenne muscular dystrophy.PloS one · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
Duchenne muscular dystrophy (DMD) is an inherited, progressive muscle wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therapy methods are being developed to replace the defective dystrophin gene or induce dystrophin production from mutant genes. Stem cell approaches are being developed to replace lost muscle cells while also bringing in new dystrophin genes. This review summarizes recent progress in the field with an emphasis on clinical applications.
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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.