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Muscle & Nerve|September 10, 2015
Respiratory motor function in individuals with centronuclear myopathiesBarbara K Smith, Markus S Renno, Meghan M Green, et al.Muscle & Nerve|September 5, 2014
Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunctionManuela Corti, Barbara K Smith, Darin J Falk, et al.Mitochondrion|November 14, 2017
Development of a novel observer reported outcome tool as the primary efficacy outcome measure for a rare disease randomized controlled trialPeter W Stacpoole, Jonathan Shuster, John L P Seamus Thompson, et al.Nutrition in Clinical Practice : Official Publication of the American Society for Parenteral and Enteral Nutrition|March 3, 2020
ASPEN Consensus Recommendations for Refeeding SyndromeJoshua S V da Silva, David S Seres, Kim Sabino, 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.The Journal of Pediatrics|April 23, 2013
B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe diseaseMelissa E Elder, Sushrusha Nayak, Shelley W Collins, et al.Molecular Genetics and Metabolism|April 11, 2025
Safety and efficacy of migalastat in adolescent patients with Fabry disease: Results from ASPIRE, a phase 3b, open-label, single-arm, 12-month clinical trial, and its open-label extensionUma Ramaswami, Esperanza Font-Montgomery, Ozlem Goker-Alpan, et al.Human Gene Therapy. Clinical Development|November 22, 2017
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe DiseaseManuela Corti, Cristina Liberati, Barbara K Smith, et al.Human Gene Therapy|April 11, 2013
Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomesBarbara K Smith, Shelley W Collins, Thomas J Conlon, et al.Human Molecular Genetics|April 27, 2011
Pompe disease gene therapyBarry J Byrne, Darin J Falk, Christina A Pacak, et al.Pageof 2