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Journal of Neuromuscular Diseases|February 20, 2025
Gross motor delays in infants and young boys with Duchenne muscular dystrophyLinda P Lowes, Natalie F Reash, Megan A Iammarino, et al.BMC Infectious Diseases|June 27, 2013
Compartmentalized, functional role of angiogenin during spotted fever group rickettsia-induced endothelial barrier dysfunction: evidence of possible mediation by host tRNA-derived small noncoding RNAsBin Gong, Yong Sun Lee, Inhan Lee, et al.Journal of Neuromuscular Diseases|August 6, 2019
AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History CohortSamiah A Al-Zaidy, Stephen J Kolb, Linda Lowes, et al.Nature Biotechnology|August 12, 2011
Astrocytes from familial and sporadic ALS patients are toxic to motor neuronsAmanda M Haidet-Phillips, Mark E Hester, Carlos J Miranda, et al.Human Gene Therapy|March 7, 2019
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb InfusionJerry R Mendell, Louis G Chicoine, Samiah A Al-Zaidy, et al.Molecular Genetics and Metabolism|August 13, 2013
Molecular and clinical characterization of the myopathic form of mitochondrial DNA depletion syndrome caused by mutations in the thymidine kinase (TK2) geneSirisak Chanprasert, Jing Wang, Shao-Wen Weng, et al.Muscle & Nerve|August 14, 2023
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trialJerry R Mendell, Zarife Sahenk, Kelly J Lehman, et al.Annals of Neurology|March 28, 2012
Evidence-based path to newborn screening for Duchenne muscular dystrophyJerry R Mendell, Chris Shilling, Nancy D Leslie, et al.Circulation|October 20, 2023
Evinacumab for Pediatric Patients With Homozygous Familial HypercholesterolemiaAlbert Wiegman, Susanne Greber-Platzer, Shazia Ali, et al.The New England Journal of Medicine|October 8, 2010
Dystrophin immunity in Duchenne's muscular dystrophyJerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, et al.Pageof 89