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TrialHuman gene therapy2013

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 et al.PubMed ↗Full text ↗Publisher ↗

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2 trials

Full record →Abstract, authors, funding and every citing paper · PMID 23570273