ReviewNeurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology2022
Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.
Review in Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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Who cites it
15 citing papers in PubMed, 29 citations in OpenAlex.
- Wearable sensors for trunk posture monitoring in Duchenne muscular dystrophy: a case study.Medical & biological engineering & computing · 2026Article
- Upper limb progression in Duchenne muscular dystrophy: Insights from a 36-month longitudinal study using the PUL 20.Journal of neuromuscular diseases · 2026Article
- Short-term daily deflazacort decreases membrane permeability and increases maximum force inAmerican journal of physiology. Cell physiology · 2025Article
- Quantitative Muscle Ultrasound: A Non-Invasive Biomarker for Monitoring Duchenne Muscular Dystrophy.Muscle & nerve · 2025Observational
- Longitudinal interaction between muscle impairments and gait pathology in growing children with Duchenne muscular dystrophy.Journal of neuroengineering and rehabilitation · 2025Observational
- Muscle weakness but also contractures contribute to the progressive gait pathology in children with Duchenne muscular dystrophy: a simulation study.Journal of neuroengineering and rehabilitation · 2025Article
- Early Cardiac Dysfunction in Duchenne Muscular Dystrophy: A Case Report and Literature Update.International journal of molecular sciences · 2025Review
- Is there a decline in speech and swallowing in Amyotrophic Lateral Sclerosis over ten years?CoDAS · 2025Article
- Longitudinal trajectories of muscle impairments in growing boys with Duchenne muscular dystrophy.PloS one · 2025Article
- Use of assistive technology to assess distal motor function in subjects with neuromuscular disease.PLOS digital health · 2025Article
- Molecular Genetic Analysis of aPharmacogenomics and personalized medicine · 2025Article
- Givinostat: First Approval.Drugs · 2024Review
- Physiological stress improves stem cell modeling of dystrophic cardiomyopathy.Disease models & mechanisms · 2024Article
- Gait classification for growing children with Duchenne muscular dystrophy.Scientific reports · 2024Article
- N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy.Skeletal muscle · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionDuchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle disease characterized by childhood-onset muscle weakness, leading to loss of motor function and premature death due to respiratory and cardiac insufficiency. DISCUSSION: In the following three and half decades, DMD kept its paradigmatic role in the field of muscle diseases, with first systematic description of disease progression with ad hoc outcome measures and the first attempts at correcting the disease-causing gene defect by several molecular targets. Clinical trials are critical for developing and evaluating new treatments for DMD.
conclusionsIn the last 20 years, research efforts converged in characterization of the disease mechanism and development of therapeutic strategies. Same effort needs to be dedicated to the development of outcome measures able to capture clinical benefit in clinical trials.
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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.