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Medicine|July 3, 2019
Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophyLindsay N Alfano, Jay S Charleston, Anne M Connolly, et al.
Muscle & Nerve|February 2, 2019
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophyAnne M Connolly, Craig M Zaidman, Paul T Golumbek, et al.
Annals of Neurology|January 23, 2015
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45Andrew R Findlay, Nicolas Wein, Yuuki Kaminoh, et al.
Human Gene Therapy|February 13, 2026
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and ManagementPerry B Shieh, Crystal Proud, Tamir Diamond, et al.
Cell Reports|October 18, 2023
ZNF692 organizes a hub specialized in 40S ribosomal subunit maturation enhancing translation in rapidly proliferating cellsM Carmen Lafita-Navarro, Yi-Heng Hao, Chunhui Jiang, et al.
Journal of Cachexia, Sarcopenia and Muscle|August 15, 2025
High-Density Lipoprotein-Associated Cholesterol Abnormalities in a Clinical Outcomes Study of Dysferlin-Deficient Limb-Girdle Muscular Dystrophy Type R2Zoe White, Laura Rufibach, Heather Gordish Dressman, et al.
Molecular Cell|January 11, 2024
Kinome-wide siRNA screen identifies a DCLK2-TBK1 oncogenic signaling axis in clear cell renal cell carcinomaLianxin Hu, Yanfeng Zhang, Lei Guo, et al.
Neurogenetics|February 29, 2012
Mutations in the satellite cell gene MEGF10 cause a recessive congenital myopathy with minicoresSteven E Boyden, Lane J Mahoney, Genri Kawahara, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|October 18, 2014
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophyJerry R Mendell, Zarife Sahenk, Vinod Malik, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|November 8, 2013
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular deliveryL G Chicoine, C L Montgomery, W G Bremer, et al.
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