Høydepunkter Fra ISTH 2020 Virtual Congress
Fase I / II-utprøving av SPK-8011: Stabil og holdbar FVIII-uttrykk i> 2 år med signifikante ABR-forbedringer i startdosekohorter etter AAV-mediert FVIII-genoverføring for hemophilia A
L. George1,2Eyster3, M. Ragni4, S. Sullivan5, B. Samelson-Jones1,2Evans3A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. Høy6
1Children's Hospital of Philadelphia, Philadelphia, USA
2Perelman School of Medicine ved University of Pennsylvania, Philadelphia, USA
3Pennsylvania State University Milton S. Hershey Medical Center, Hershey, USA
4University of Pittsburgh Medical Center and Hemophilia Center of Western Pennsylvania, Department of Medicine, Pittsburgh, USA
5Mississippi Center for Advanced Medicine, Madison, USA
6Spark Therapeutics, Philadelphia, USA
L. George1,2Eyster3, M. Ragni4, S. Sullivan5, B. Samelson-Jones1,2Evans3A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. Høy6
1Children's Hospital of Philadelphia, Philadelphia, USA
2Perelman School of Medicine ved University of Pennsylvania, Philadelphia, USA
3Pennsylvania State University Milton S. Hershey Medical Center, Hershey, USA
4University of Pittsburgh Medical Center and Hemophilia Center of Western Pennsylvania, Department of Medicine, Pittsburgh, USA
5Mississippi Center for Advanced Medicine, Madison, USA
6Spark Therapeutics, Philadelphia, USA
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