Duchenne Muscular Dystrophy — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide.
Wang Biaobiao et al. — Biomaterials (1 June 2026)
https://pubmed.ncbi.nlm.nih.gov/41506143/
- 2.
Machine learning for site risk prediction in clinical trials: development, external validation, and operational application in site qualification.
Yang Zhiwen — International journal of medical informatics (1 May 2026)
https://pubmed.ncbi.nlm.nih.gov/41741318/
- 3.
Myofibre Density Reveals a Critical Threshold Around Age 6 in Steroid-Naïve Duchenne Muscular Dystrophy: A Retrospective Observational Study.
Yamakado Tetsuhiro et al. — Neuropathology and applied neurobiology (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41848212/
- 4.
Longitudinal ankle range of motion and functional decline in Duchenne muscular dystrophy.
Fleerakkers Evelien et al. — Neuromuscular disorders : NMD (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41775572/
- 5.
Effects of Bisphosphonates on Bone Micro-Architecture of Children With Duchenne Muscular Dystrophy: A Prospective Comparative Study.
Wang Songqi et al. — Journal of cachexia, sarcopenia and muscle (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41749413/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
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
- 2.
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
Recruiting — University of Kansas Medical Center
https://clinicaltrials.gov/study/NCT06847282
- 3.
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Recruiting — Phase 2 — Satellos Bioscience, Inc.
https://clinicaltrials.gov/study/NCT07287189
- 4.
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Recruiting — Phase 2 — NS Pharma, Inc.
https://clinicaltrials.gov/study/NCT05996003
- 5.
A Study of Bleeding and Treatment in Participants With Von Willebrand Disease
Recruiting — Hemab ApS
https://clinicaltrials.gov/study/NCT06610201
- 6.
The Efemoral Vascular Scaffold System (EVSS) for the Treatment of Patients with Symptomatic Peripheral Vascular Disease from Stenosis or Occlusion of the Femoropopliteal Artery
Recruiting — Na — Efemoral Medical, Inc.
https://clinicaltrials.gov/study/NCT04584632
- 7.
First In Human Study to Assess Safety and Efficacy of the ChampioNIR™ Drug Eluting Peripheral Stent in the Treatment of Patients With Superficial Femoral Artery Disease and/or Proximal Popliteal Artery Disease
Recruiting — Na — Medinol Ltd.
https://clinicaltrials.gov/study/NCT06410313
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
Although peptide-based delivery strategies show promise for muscle and heart diseases, delivery of biotherapeutics to both skeletal and cardiac muscles remains challenging. Here, we identified a muscle-homing peptide (BV2) against blood vessel epicardial substance (BVES) by phage display. BV2 shows high binding affinity to BVES and is internalized primarily via caveolae-mediated endocytosis. Importantly, BV2 enables efficient delivery of Duchenne Muscular Dystrophy (DMD) phosphorodiamidate morpholino oligomer (PMO), mCherry protein and exosomes to skeletal muscle and heart in vivo. BV2-mCherry protein and BV2-E31R anti-myostatin peptide were effectively delivered to muscle layers when microneedles loaded with these biotherapeutics were implanted on hindlimbs of mice. Muscle mass and myofiber size also significantly increased in muscle atrophy mice grafted with BV2-E31R microneedles. Moreover, significantly enhanced restoration of dystrophin protein was achieved in peripheral and cardiac muscles of dystrophin-deficient mdx and dystrophin/utrophin double-knockout mice when exosomes simultaneously modified with BV2 and PMO. These findings highlight the potency of BV2 in directing targeted delivery of diverse biotherapeutics to muscle and heart, thus providing an effective tool for DMD and other muscular and cardiac disorders.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.