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Annals of Neurology|February 27, 2013
LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophyKevin M Flanigan, Ermelinda Ceco, Kay-Marie Lamar, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|March 11, 2017
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional OutcomesJerry R Mendell, Zarife Sahenk, Samiah Al-Zaidy, et al.Journal of Neuromuscular Diseases|March 13, 2023
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)Richard S Finkel, Basil T Darras, Jerry R Mendell, et al.Neuromuscular Disorders : NMD|December 24, 2023
Continued safety and long-term effectiveness of onasemnogene abeparvovec in OhioMegan A Waldrop, Shannon Chagat, Michael Storey, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|October 23, 2013
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogatesLouis G Chicoine, Louise R Rodino-Klapac, Guohong Shao, et al.The Journal of Urology|August 12, 2005
Intensive lifestyle changes may affect the progression of prostate cancerDean Ornish, Gerdi Weidner, William R Fair, et al.JAMA Neurology|June 16, 2020
Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled TrialJerry R Mendell, Zarife Sahenk, Kelly Lehman, et al.Molecular Medicine (Cambridge, Mass.)|January 27, 2011
Peptide-based inhibition of NF-κB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophyJennifer M Peterson, William Kline, Benjamin D Canan, et al.Science Advances|July 20, 2022
Leveraging gene therapy to achieve long-term continuous or controllable expression of biotherapeuticsTimothy P Cripe, Brian Hutzen, Mark A Currier, et al.Nature Medicine|October 5, 2021
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissueGretchen Thomsen, Arthur H M Burghes, Caroline Hsieh, et al.Pageof 89