Duchenne Muscular Dystrophy — Research Summary
Printed from RareWays (rareways.com.au) on 27 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.
Moriyama Hidenori et al. — RNA biology (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/42324661/
- 2.
Comment on "Radiosensitivity and delayed radiation-induced nucleo-shuttling of the ATM protein in fibroblasts from Duchenne muscular dystrophy expressing residual dystrophin".
Sati Deepti M et al. — Journal of the neurological sciences (15 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42107324/
- 3.
Reply to the Letter of Editor addressed by D.M. Sati et al., about our article entitled "Radiosensitivity and Delayed Radiation-Induced Nucleo-shuttling of the ATM Protein in Fibroblasts from Duchenne Muscular Dystrophy Expressing Residual Dystrophin".
Foray Nicolas et al. — Journal of the neurological sciences (15 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42102643/
- 4.
Duchenne Muscular Dystrophy and Delandistrogene Moxeparvovec Gene Therapy in Children: A Systematic Review and Meta-Analysis.
Antonello Breno Bopp et al. — Neurology. Genetics (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42396397/
- 5.
Basal Energetics and Phosphocreatine Recovery Kinetics in Ambulatory Boys With Duchenne Muscular Dystrophy.
Awale Pratiksha P et al. — NMR in biomedicine (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42324864/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Recruiting — Phase 2 — NS Pharma, Inc.
https://clinicaltrials.gov/study/NCT05996003
- 2.
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Recruiting — Phase 2 — Satellos Bioscience, Inc.
https://clinicaltrials.gov/study/NCT07287189
- 3.
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Recruiting — Phase 3 — Solid Biosciences Inc.
https://clinicaltrials.gov/study/NCT07160634
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a genetic condition causing progressive muscle weakness, mainly affecting boys. It is caused by a missing protein called dystrophin. Research into gene therapy and other treatments has advanced rapidly in recent years.
Most Recent Research
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disorder caused by dystrophin deficiency. Antisense oligonucleotide (ASO)-mediated exon skipping has emerged as a cornerstone of DMD therapy to restore dystrophin expression. This review provides a comprehensive overview of the four FDA-approved ASO therapies - eteplirsen, golodirsen, viltolarsen, and casimersen - tracing their journey from pivotal clinical trials to post-marketing updates. While the development and clinical evaluation of these agents have established a pioneering framework for rare genetic diseases, they have also highlighted critical challenges. These include complexities in clinical trial design, discrepancies between preclinical efficacy and clinical outcomes, real-world burdens, and limited patient eligibility. Furthermore, the FDA's accelerated approval of these therapies based on limited clinical data remains a subject of ongoing debate. Confirmatory trials to verify clinical efficacy and long-term follow-up studies are actively underway. Concurrently, intensive research is focused on developing next-generation ASOs to achieve enhanced therapeutic efficacy and definitive clinical outcomes. Elucidating the trajectory of research and development in this field offers profound insights for shaping future therapeutic strategies in rare diseases.
Common Questions
What is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy is a genetic condition causing progressive muscle weakness, mainly affecting boys. It is caused by a missing protein called dystrophin. Research into gene therapy and other treatments has advanced rapidly in recent years.
How many clinical trials are available for Duchenne Muscular Dystrophy?
RareWays currently indexes 348 clinical trials for Duchenne Muscular Dystrophy, of which 69 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Duchenne Muscular Dystrophy come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.