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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.Neurology|February 27, 2001
Randomized controlled trial of IVIg in untreated chronic inflammatory demyelinating polyradiculoneuropathyJ R Mendell, R J Barohn, M L Freimer, et al.Elife|October 8, 2015
Precise let-7 expression levels balance organ regeneration against tumor suppressionLinwei Wu, Liem H Nguyen, Kejin Zhou, et al.Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.Cancer Medicine|November 21, 2019
A community-based lung cancer rapid tissue donation protocol provides high-quality drug-resistant specimens for proteogenomic analysesTheresa A Boyle, Gwendolyn P Quinn, Matthew B Schabath, et al.Human Mutation|October 6, 2011
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD geneKevin M Flanigan, Diane M Dunn, Andrew von Niederhausern, et al.Annals of Neurology|August 3, 2013
Eteplirsen for the treatment of Duchenne muscular dystrophyJerry R Mendell, Louise R Rodino-Klapac, Zarife Sahenk, et al.Nature Cancer|September 28, 2020
eIF5B drives integrated stress response-dependent translation of PD-L1 in lung cancerShruthy Suresh, BeiBei Chen, Jingfei Zhu, et al.Neurology and Therapy|January 10, 2026
Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK TrialJerry R Mendell, Francesco Muntoni, Craig M McDonald, et al.Pageof 89