Højdepunkter Fra ISTH 2020 Virtual Congress
Fase I / II-forsøg med SPK-8011: Stabil og holdbar FVIII-ekspression i> 2 år med væsentlige ABR-forbedringer i startdosekohorter efter AAV-medieret FVIII-genoverførsel til hæmofili A
L. George1,2Eyster3, M. Ragni4Sullivan5, B. Samelson-Jones1,2Evans3A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. Høj6
1Børnehospital i 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. Ragni4Sullivan5, B. Samelson-Jones1,2Evans3A. MacDougall6, M. Curran6, S. Tompkins6, K. Wachtel6Takefman6, K. Reape6, F. Mingozzi6, P. Monahan6, X. Anguela6, K. Høj6
1Børnehospital i 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
RETTET INDHOLD
Interaktive webinarer
Interaktive webinarer
Podcasts