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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

Research output: Contribution to journal › Article › peer-review

137 Citations (Scopus)

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