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Matthew S. Evans
Witold B. Rybka
Stacy E. Croteau
Huyen Tran
John E.J. Rasko
Kristen Jaworski
Amy Macdougall
Savina Jaeger
Federico Mingozzi
Tiffany Chang
Gallia Levy
Australian Centre For Blood Diseases, Monash University, Melbourne, Australia
Penn State Health Milton S. Hershey Medical Center, Hershey, PA
Boston Children's Hospital, Boston, MA
Spark Therapeutics, Philadelphia, PA
Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, and the Gene and Stem Cell Therapy Program, Centenary Institute, Faculty of Medicine and Health, University of Sydney, Camperdown, Australia
Australia
United States
N/A
N/A
N/A
Alanine transaminase
Interferon
FVIII
N/A
Randomized
Open Label
Multicenteric
Dose Escalation
I/II
N/A
N/A
N/A