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Pediatric Pulmonology|February 15, 2024
Longitudinal changes of swallowing safety and efficiency in infants with spinal muscular atrophy who received disease modifying therapiesCarmen Leon-Astudillo, Olivia Brooks, Stephanie M Salabarria, et al.Muscle & Nerve|September 10, 2015
Respiratory motor function in individuals with centronuclear myopathiesBarbara K Smith, Markus S Renno, Meghan M Green, et al.Annals of Translational Medicine|August 9, 2019
Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapyBarry J Byrne, David D Fuller, Barbara K Smith, et al.Human Gene Therapy|October 30, 2025
Enhancing AAV9-UFμDys1 Gene Therapy Efficacy Through Immunosuppression in Mice with Pre-Existing Immunity and Enabling Redosing Strategies for Duchenne Muscular DystrophyMadhurima Saha, Radhika Bhake, Craig A Meyers, et al.Experimental Neurology|July 26, 2016
Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticityBarbara K Smith, A Daniel Martin, Lee Ann Lawson, et al.JIMD Reports|April 15, 2018
Reduced Muscle Strength in Barth Syndrome May Be Improved by Resistance Exercise Training: A Pilot StudyAdam J Bittel, Kathryn L Bohnert, Dominic N Reeds, et al.Journal of Neuromuscular Diseases|March 19, 2026
Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophyCarmen Leon-Astudillo, Stephanie M Salabarria, Christina B Chadwick, et al.Human Gene Therapy. Clinical Development|September 22, 2015
Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical PlanningManuela Corti, Brian Cleaver, Nathalie Clément, et al.JIMD Reports|November 12, 2021
Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot studyKathryn L Bohnert, Grace Ditzenberger, Adam J Bittel, et al.Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|September 14, 2024
Neurological glycogen storage diseases and emerging therapeuticsMatthieu Colpaert, Pankaj K Singh, Katherine J Donohue, et al.Pageof 6