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Muscle & Nerve|April 5, 2013
Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulationXiomara Q Rosales, Vinod Malik, Amita Sneh, et al.
Human Molecular Genetics|July 19, 2013
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD modelLouise R Rodino-Klapac, Paul M L Janssen, Kimberly M Shontz, et al.
Neurology|May 13, 2018
Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin productionJay S Charleston, Frederick J Schnell, Johannes Dworzak, et al.
Annals of Clinical and Translational Neurology|March 28, 2015
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal ModelsPatricia C Sondergaard, Danielle A Griffin, Eric R Pozsgai, et al.
Molecular Genetics & Genomic Medicine|March 25, 2015
A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican HispanicsSamiah A Al-Zaidy, Vinod Malik, Kelley Kneile, et al.
Annals of Clinical and Translational Neurology|April 28, 2025
Validity and Reliability of Clinical and Patient-Reported Outcomes in Multisystem Proteinopathy 1Lindsay N Alfano, Megan A Iammarino, Natalie F Reash, et al.
Cancer Cell|November 16, 2005
Dystrophin glycoprotein complex dysfunction: a regulatory link between muscular dystrophy and cancer cachexiaSwarnali Acharyya, Matthew E R Butchbach, Zarife Sahenk, 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.
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