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Annals of Neurology|August 4, 2023
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR)Craig M Zaidman, Crystal M Proud, Craig M McDonald, et al.
Annals of Neurology|October 3, 2009
Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteinsJerry R Mendell, Louise R Rodino-Klapac, Xiomara Rosales-Quintero, 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.
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.
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 Therapy. Methods & Clinical Development|August 4, 2018
An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus MacaqueRui Xu, Ying Jia, Deborah A Zygmunt, 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.
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.
JCI Insight|November 16, 2018
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHDCarlee R Giesige, Lindsay M Wallace, Kristin N Heller, et al.
Annals of Neurology|October 30, 2010
Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2DJerry R Mendell, Louise R Rodino-Klapac, Xiomara Q Rosales, et al.
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