New trials, publications, preprints and grants on this target, month by month.
Publication
Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials.
29 Oct 2026· Journal of medical genetics· PUBMED
Publication
Teaching NeuroImage: Somatic DMD Gene Mosaicism Causing Hemiatrophy in an Adult Man With Duchenne Muscular Dystrophy.
26 Oct 2026· Neurology· PUBMED
Publication
Statistical correlation between strength and mini-dystrophin expression in skeletal muscle after an rAAV9-mini-dystrophin gene therapy candidate in DMDmdx rats.
17 Oct 2026· Gene therapy· PUBMED
Publication
Patient-derived teratomas as a humanized platform for dystrophin restoration by ex vivo cell therapy and in vivo base editing.
1 Oct 2026· Experimental & molecular medicine· PUBMED
Publication
From molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.
22 Sept 2026· Frontiers in pharmacology· PUBMED
Publication
Mutational Spectrum of DMD Gene Variants and Cascade Screening Outcomes in the Central-Eastern Black Sea Region of Türkiye: A Single-Centre Retrospective Study.
22 Sept 2026· Molecular biology reports· PUBMED
Publication
Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.
10 Sept 2026· Molecular therapy : the journal of the American Society of Gene Therapy· PUBMED
Publication
Proteomics of the skeletal muscle dystrophin complex.
8 Sept 2026· Advances in clinical chemistry· PUBMED
Trial
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
1 Sept 2026· PHASE3 · RECRUITING· CTGOV
Publication
Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.
28 Aug 2026· Disease models & mechanisms· PUBMED
Publication
Exon-skipping therapies for DMD in Kazakhstan: Progress and challenges.
27 Aug 2026· Journal of neuromuscular diseases· PUBMED
Publication
Characterization of Dystrophin-Related Syndromes: Carriers, DMD, and BMD.
26 Aug 2026· Muscles (Basel, Switzerland)· PUBMED
Publication
Beyond dystrophin: cell therapy for Duchenne muscular dystrophy.
22 Aug 2026· Lancet (London, England)· PUBMED
Publication
Evaluation of antibody reactivity to dystrophin products: does Dp40 really exist?
4 Aug 2026· Neuromuscular disorders : NMD· PUBMED
Publication
Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping.
3 Aug 2026· Molecular therapy. Nucleic acids· PUBMED
Publication
Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.
31 Jul 2026· Genome medicine· PUBMED
Trial
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
28 Jul 2026· PHASE3 · RECRUITING· CTIS
Publication
A high-fat, high-sucrose diet exacerbates muscle and metabolic pathology and undermines glucocorticoid efficacy in dystrophin-deficient mice.
1 Jul 2026· The Journal of physiology· PUBMED
Publication
Dystrophin-Deficient Muscular Dystrophy in a Jack Russell Terrier With a Large Deletion in the Canine DMD Gene.
1 Jul 2026· Veterinary medicine and science· PUBMED
Publication
Novel DMD Frameshift Variant (p.Leu2017Profs*5) in Spectrin-Like Repeat 16 Expands the Mutational Spectrum of DMD.
1 Jul 2026· Molecular genetics & genomic medicine· PUBMED
Publication
Calcium handling properties and arrhythmia vulnerability of cardiomyocytes from dystrophin-deficient mdx mice.
22 Jun 2026· Cell calcium· PUBMED
Publication
Muscle-targeted extracellular vesicles for full-length dystrophin mRNA therapy in Duchenne muscular dystrophy.
11 Jun 2026· Nature biomedical engineering· PUBMED
Publication
Exploring Dystrophin Expression and Mutations in the DMD and Dystrophin-Glycoprotein Complex Genes as Prognostic Factors in Leiomyosarcomas.
11 Jun 2026· International journal of molecular sciences· PUBMED
Publication
Gene editing restores full-length dystrophin and affects iron homeostasis in hiPSC-derived cardiomyocytes from Becker Muscular Dystrophy patients.
5 Jun 2026· Journal of neuromuscular diseases· PUBMED
Publication
Microbiota dysbiosis influences immune system and muscle pathophysiology of dystrophin deficient mice.
3 Jun 2026· EMBO molecular medicine· PUBMED
Preprint
Stress unmasks impaired endocrine coordination of glucose metabolism in dystrophin deficiency.
27 May 2026· bioRxiv : the preprint server for biology· PUBMED
Publication
A female manifesting carrier of DMD with exon 45 deletion: a case report.
25 May 2026· Translational pediatrics· PUBMED
Publication
Reply to the letter of editor addressed by Thulamy Raman D. 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".
23 May 2026· Journal of the neurological sciences· PUBMED
Publication
Comment on "Radiosensitivity and delayed radiation-induced nucleo-shuttling of the ATM protein in fibroblasts from Duchenne muscular dystrophy expressing residual dystrophin".
22 May 2026· Journal of the neurological sciences· PUBMED
Publication
Quantification of a serum titin fragment reflects dystrophin restoration in mdx mice and disease severity in patients with dystrophinopathies.
20 May 2026· Journal of neuromuscular diseases· PUBMED