ReviewCurrent neurology and neuroscience reports2020
Update on Muscular Dystrophies with Focus on Novel Treatments and Biomarkers.
Review in Current neurology and neuroscience reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 2 syntheses or guidelines pooled it, 38 citations in OpenAlex.
- Dyslipidemia in Muscular Dystrophy: A Systematic Review and Meta-Analysis.Journal of neuromuscular diseases · 2023Pooled it
- Using MRI to quantify skeletal muscle pathology in Duchenne muscular dystrophy: A systematic mapping review.Muscle & nerve · 2021Pooled it
- L-Arginine Activates the Neuregulin-1/ErbB Receptor Signaling Pathway and Increases Utrophin mRNA Levels in C2C12 Cells.Biochemistry research international · 2025Article
- Review
- Plumbing mysterious RNAs in "dark genome" for the conquest of human diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Difficulties in social cognitive functioning among pediatric patients with muscular dystrophies.Frontiers in psychology · 2023Article
- Development and Validation of a Western Blot Method to Quantify Mini-Dystrophin in Human Skeletal Muscle Biopsies.The AAPS journal · 2022Article
- Sphingolipids mediate lipotoxicity in muscular dystrophies.Life medicine · 2022Article
- Nanomedicine for Treating Muscle Dystrophies: Opportunities, Challenges, and Future Perspectives.International journal of molecular sciences · 2022Review
- Lysosomes and the pathogenesis of merosin-deficient congenital muscular dystrophy.Human molecular genetics · 2022Article
- The Donnan-dominated resting state of skeletal muscle fibers contributes to resilience and longevity in dystrophic fibers.The Journal of general physiology · 2022Article
- Perspectives on hiPSC-Derived Muscle Cells as Drug Discovery Models for Muscular Dystrophies.International journal of molecular sciences · 2021Review
- Casimersen: First Approval.Drugs · 2021Review
- BioengineeredJournal of tissue engineeringReview
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purpose of reviewMuscular dystrophies are a heterogeneous group of inherited muscular disorders characterized by progressive muscle weakness and in many cases cardiac and respiratory muscle involvement. Historically, these disorders are considered incurable with grave prognoses. The genes responsible for most muscular dystrophies are known, and early diagnosis is achievable with proper clinical recognition and advanced genetic testing. This article reviews recent advances in the development of novel treatments and biomarkers in the realm of muscular dystrophies commonly encountered in pediatric population. RECENT
findingsThe therapeutic landscape of muscular dystrophies has changed with the development of new approved treatments for Duchenne muscular dystrophy (DMD), the most common and severe muscular dystrophy. This has paved the way for the development of novel therapeutic strategies for not only DMD but also other muscular dystrophies. This article reviews recent advances in the development of novel treatments and biomarkers in the realm of muscular dystrophies commonly encountered in pediatric population.
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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 Socratic graph.