Phase I/II Trial of Adeno-associated Virus Acid-alpha-Glucosidase (AAV-GAA) Diaphragm Gene Therapy for Ventilatory Failure in Pompe Disease
Barry J. Byrne(University of Florida), Shelley Collins(University of Florida), Thomas J. Conlon(Adelson Foundation), Barbara K. Smith(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), Cathryn Mah(Anna Needs Neuroblastoma Answers)
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