Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity - Smith, Barbara K.; Martin, A. Daniel; Lawson, Lee Ann; Vernot, Valerie; Marcus, Jordan; Islam, Saleem; Shafi, Nadeem; Corti, Manuela; Collins, Shelley W.; Byrne, Barry J. (Experimental neurology, 2017)
Keywords: Adenoviridae - genetics; Adenoviridae - metabolism; Adolescent; alpha-Glucosidases - genetics; alpha-Glucosidases - therapeutic use; Child; Child, Preschool; Diaphragm; Diaphragm - physiology; Electromyography; Exercise Therapy; Female; Gene therapy; Genetic Therapy; Glycogen Storage Disease Type II - complications; Glycogen Storage Disease Type II - diagnostic imaging; Glycogen Storage Disease Type II - genetics; Glycogen Storage Disease Type II - therapy; Humans; Magnetic Resonance Imaging; Male; Muscle, Skeletal - physiopathology; Pompe disease; Prospective Studies; Respiratory Insufficiency - diagnostic imaging; Respiratory Insufficiency - etiology; Respiratory Insufficiency - therapy; Treatment Outcome; Ventilatory insufficiency; Pompe disease; MIP; MRI; Ventilatory insufficiency; ERT; GAA; IMST; LC; MV; Diaphragm; Gene therapy