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(University of Florida), Barry J. Byrne(University of Florida), Cathryn Mah(Anna Needs Neuroblastoma Answers), Shelley Collins(University of Florida), Thomas J. Conlon(Adelson Foundation), David D. Fuller(Brenau University), Brian Cleaver(University of Florida), Nicole Dobjia(University of Florida), Dawn Phillips(ProterixBio (United States)), Anatole D. Martin(University of Florida Health), Lee Ann Lawson(University of Florida), Saleem Islam(University of Florida), Nathalie Clément(University of Florida)
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