Last Updated: 09/26/2026
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Found 145 publications
Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.
Journal: The New England journal of medicine
Published: July 22, 2026
Histological, functional, and behavioral comparison of three Duchenne muscular dystrophy mouse models.
Journal: Neuromuscular disorders : NMD
Published: June 26, 2026
Plasma EV Proteomics Identifies ECM Remodeling and Inflammatory Proteins LUM and C7 as Candidate Biomarkers in FSHD.
Journal: Annals of clinical and translational neurology
Published: April 27, 2026
U7snRNA-mediated skipping of intron-derived pseudoexons restores full-length DMD expression in patient-derived cell lines.
Journal: Molecular therapy. Advances
Published: January 14, 2026
Unraveling the spatial landscape of dystrophinopathies: a transcriptomic approach to Becker and Duchenne muscular dystrophies.
Journal: The Journal of pathology
Published: November 10, 2025
Gene x environment interaction analysis confirms genetic modifier effects on steroid efficacy via TGF-β pathway in Duchenne muscular dystrophy.
Journal: European journal of human genetics : EJHG
Published: September 23, 2025
ALT/CK Ratio as an Early Marker of Liver Injury After Gene Therapy in Duchenne Muscular Dystrophy.
Journal: Pediatrics
Published: September 04, 2025
Generation of an induced pluripotent stem cell line (NCHi026-A) from a patient with a partial deletion of exon 55 in the DMD gene.
Journal: Stem cell research
Published: August 20, 2025
Predicting subtypes of glycogen storage disease type IV: Challenges of hepatic subtypes and genotype-phenotype correlation.
Journal: Molecular genetics and metabolism
Published: August 14, 2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy.
Journal: Neuromuscular disorders : NMD
Published: June 17, 2025
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK.
Journal: European journal of medical genetics
Published: May 15, 2025
Like father, like son: RNA-sequencing from a 30-year-old muscle biopsy identifies a novel splice variant in ACTA1 as the cause of an attenuated nemaline myopathy phenotype.
Journal: Neuromuscular disorders : NMD
Published: February 26, 2025
Last Updated: 09/26/2026