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.
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T. Conlon | B. Byrne | D. Fuller | N. Clément | C. Mah | Barbara K. Smith | S. Islam | L. Lawson | S. Collins | B. Cleaver | Dawn Phillips | Anatole D. Martin | Nicole Dobjia | Lee Ann Lawson