Gainesville / Teng / Chen / Mendelsohn / Boston / Baltimore / Bercury / /
Company
J.M. (2009) Worldwide / A. and Koeberl D.D. / Human Applications Laboratory / C.A.P. D.D. / B.L. and Koeberl D.D. / Florida Powell Gene Therapy Center Vector Core Laboratory / UF Toxicology Core Laboratory / Oxford University Press / Wilson / Donnelly / Fuller D.D. / Y.T. and Koeberl D.D. / IgG / Young S.P. Li / Young S.P. / Winkel L.P. / Van der Beek N.A. / Germain S.A. / Suzuki / Smith S.A. Li / Rosenberg A.S. / /
Country
United States / /
Currency
pence / / /
Event
FDA Phase / /
Facility
Hospital Boston / University of Florida / Human Applications Laboratory / The Johns Hopkins University / University of Florida Powell Gene Therapy Center Vector Core Laboratory / UF Toxicology Core Laboratory / /
IndustryTerm
viral vector delivery systems / suppression protocols / invaluable tool / care for the treatment of Pompe disease / therapy for lysosomal storage disease / treatment of Pompe disease / therapy for Pompe disease / injection site / tolerance protocols / cardiac magnetic resonance imaging / treatment of neurological disorders / treatment of the high rate of respiratory failure / therapy for hemophilia / organ systems / gene therapy protocols / transgene product / therapy of brain diseases / /
American Society of Gene and Cell Therapy / The Johns Hopkins University / AAV2/ 8-LSP-hGAApA administration / Systemic administration / 2Powell Gene Therapy Center / Oxford University / American Society of Gene Therapy / Direct administration / Targeted administration / Department of Pediatrics / University of Florida / /
Person
Shelley W. Collins / Darin J. Falk / Stacy L. Porvasnik / Drs Kai Qiu / Cathryn S. Mah / Brian D. Cleaver / David D. Fuller / Barry J. Byrne / Van Doorn / Van den Hout / Paul Reier / Melissa E. Elder / De Klerk / Malik / V / Christina A. Pacak / Van Hove / J. Gene Med / Saleem Islam / Barbara K. Smith / Nina Raben / Roland W. Herzog / Nathalie Clement / Mai K. Elmallah / Anatole Martin / Lee Ann Lawson / Soustek / Thomas J. Conlon / Foley / / /
Position
D.J. / Author / King / Cao / /
Product
cyclophosphamide / Myozyme / POMPE PATIENTS WITH / /
ProvinceOrState
Bali / Y.T. / New Jersey / S.K. / Maryland / South Dakota / Florida / /