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 outcomes

Barbara K. Smith, Shelley W. Collins, Thomas J. Conlon, Cathryn S. Mah, Lee Ann Lawson, Anatole D. Martin, David D. Fuller, Brian D. Cleaver, Nathalie Clément, Dawn Phillips, Saleem Islam, Nicole Dobjia, Barry J. Byrne

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