Duchenne muscular dystrophyNews & Research
17 curated articles for Duchenne muscular dystrophy — FDA updates, peer-reviewed research, clinical-trial milestones, and sponsor press releases. Sorted newest-first.
- Mol Ther Nucleic Acids Jun 16, 2026
The post-pandemic pivot: mRNA therapeutics enter the chronic rare disease arena
Published in Mol Ther Nucleic Acids. Giangrande PH et al.
Why it matters: Recent peer-reviewed research relevant to rare disease diagnosis or treatment.
- Molecular therapy : the journal of the American Society of Gene Therapy Apr 9, 2026
CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disorder caused by loss of dystrophin. Upregulating utrophin, a dystrophin paralog, is a promising gene therapy approach. Here, we present a CRISPR-Cas9-based strategy to enhance utrophin expression by disrupting repressor binding sites. Us...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Current opinion in pediatrics Apr 1, 2026
Treatment advances for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a severe X-linked muscle wasting disease with childhood onset that leads to loss of ambulation in the teenage years and mortality in the second or third decade of life. Prior to 10 years ago, disease-modifying pharmacologic intervention was limited to glucocortic...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- European journal of translational myology Mar 31, 2026
Serum biomarkers in Duchenne muscular dystrophy: a leap toward precision diagnostics.
I was interested to read the article by Dowling et al.1 in the European Journal of Translational Myology, where the authors provide a comprehensive review of serum biomarkers for Duchenne Muscular Dystrophy (DMD), a devastating early-onset muscle wasting disease. The emphasis of research on liquid b...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Genes Mar 28, 2026
Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations.
Background/Objectives : Recombinant adeno-associated virus (AAV) vectors have revolutionized gene therapy for monogenic diseases such as Duchenne muscular dystrophy (DMD). However, high systemic doses required for muscle transduction cause a spectrum of toxicities ranging from transient hepatic infl...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Expert Opin Drug Discov Mar 26, 2026
The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery
Published in Expert Opin Drug Discov. Ruchi R et al.
Why it matters: Recent peer-reviewed research relevant to rare disease diagnosis or treatment.
- Autophagy Mar 25, 2026
Muscle meets Lysosomes: emerging strategies in muscular dystrophy.
Duchenne muscular dystrophy (DMD) is caused by the loss of DMD (dystrophin), leading to sarcolemmal fragility and progressive muscle degeneration. Although adeno-associated viral (AAV) microdystrophin ( µDMD ) therapies have advanced clinically, their benefits remain partial, highlighting the n...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Journal of pediatric urology Mar 18, 2026
Bladder dysfunction in Duchenne muscular dystrophy: A narrative review.
Duchenne muscular dystrophy (DMD), a severe, progressive disease, causes loss of function of skeletal muscles. Urological symptoms in DMD include detrusor overactivity, bladder hypocontractility, urinary incontinence, detrusor-sphincter dyssynergia and urolithiasis. This narrative review summarises ...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Pediatric cardiology Mar 6, 2026
Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy.
This study seeks to understand cardiac medication use in a large cohort of males with Duchenne Muscular Dystrophy (DMD) followed prospectively with focus on current practices and adherence to consensus directed medical therapy (CDMT). DMD patients have been enrolled in the Advanced Cardiac Therapies...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Immunometabolism (Cobham, Surrey) Mar 6, 2026
From lipid switch to tissue repair: how resolvins reprogram macrophage polarization and function.
Specialized pro-resolving mediators (SPMs) derived from docosahexaenoic acid (DHA), particularly D-series resolvins (RvD1, RvD2, RvD3, and RvD5), function to terminate inflammation while preserving host defense. They are synthesized from DHA by lipoxygenases and act through G‑protein‑c...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Journal of neuromuscular diseases Mar 5, 2026
Antisense RNA therapies for muscular dystrophies.
Inherited muscular dystrophies are a heterogeneous group of diseases, caused by different types of genetic mutations. RNA therapies, and particularly antisense oligonucleotides, offer a palette of therapeutic strategies to either reduce the production of harmful proteins or to restore or increase pr...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- NotableFDA Feb 10, 2026
FDA Grants Orphan Drug Designation for Novel Duchenne Therapy
A novel antisense oligonucleotide targeting exon 51 skipping received orphan drug designation for Duchenne muscular dystrophy.
Why it matters: Expands treatment options for the most common Duchenne mutation type.
- Journal of cachexia, sarcopenia and muscle Feb 4, 2026
Direct AMPK Activation Confers Mutation-Independent Therapeutic Benefit in Duchenne Muscular Dystrophy.
Duchenne muscular dystrophy (DMD) is a severe, life-limiting neuromuscular disorder (NMD) characterized by progressive muscle wasting and mitochondrial dysfunction. Although gene therapies offer promise, even those already approved by regulatory agencies, their use remains constrained by mutation sp...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Cell reports Jan 30, 2026
Muscle satellite cell editing by LNP-CRISPR-Cas9 to resist muscle injury.
Muscle satellite cells are essential for skeletal muscle regeneration and represent an attractive therapeutic target for gene delivery in Duchenne muscular dystrophy (DMD). However, efficient in vivo transduction of these cells has remained challenging. Here, we demonstrate that lipid nanoparticle (...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- Journal of the Endocrine Society Jan 9, 2026
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone.
Adrenal suppression is an iatrogenic form of adrenal insufficiency that occurs secondary to exogenous glucocorticoids (GCs) and is a documented cause of premature mortality among individuals with Duchenne muscular dystrophy (DMD). Adrenal suppression in DMD necessitates awareness and careful managem...
Why it matters: Recent peer-reviewed research on Duchenne muscular dystrophy that may be relevant for patients and caregivers.
- BreakingHHS Aug 15, 2025
Newborn Screening Expands to Include More Rare Diseases
The Advisory Committee recommended adding Duchenne MD and Pompe disease to the Recommended Uniform Screening Panel.
Why it matters: Earlier detection means earlier treatment. Could transform outcomes for thousands of newborns.
- BreakingFDA Jun 20, 2025
Elevidys Gene Therapy Receives Full FDA Approval for Duchenne MD
Sarepta Therapeutics received full FDA approval for Elevidys for ambulatory Duchenne muscular dystrophy patients aged 4-5.
Why it matters: First gene therapy for Duchenne with full approval. Sets precedent for rare neuromuscular diseases.
More on Duchenne muscular dystrophy
Articles aggregated from peer-reviewed journals (PubMed), sponsor press releases, SEC 8-K filings, and FDA announcements. Original-source links are preserved on each article page. Editorial tags (Breaking / Notable / Update) reflect UniteRare's curation-time priority assessment.