Last Updated: 09/26/2026
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Found 40 publications
Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD.
Journal: The New England journal of medicine
Published: July 22, 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
Quantification of Dystrophin Expression by Capillary Immunoassay.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: October 01, 2025
Preparation, Sectioning, and Histochemical Staining of Muscle Tissue.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: October 01, 2025
Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium.
Journal: Muscle & nerve
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
Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone.
Journal: Journal of the Endocrine Society
Published: June 18, 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
Immune-Mediated Megaconial Myopathy: A Novel Subtype of Autoimmune Myopathy.
Journal: Neurology
Published: October 30, 2024
Heterozygous loss-of-function variants in SPTAN1 cause an early childhood onset distal myopathy.
Journal: Genetics in medicine : official journal of the American College of Medical Genetics
Published: October 15, 2024
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.
Journal: Journal of neuromuscular diseases
Published: March 10, 2024
Last Updated: 09/26/2026