NBDF showcases a wide array of findings from the latest in bleeding disorders research at our annual Bleeding Disorders Conference.

Efficacy, safety and pharmacokinetics of once-weekly prophylactic emicizumab (ACE910) in pediatric persons (<12 years) with hemophilia A with inhibitors: interim analysis of single-arm, multicenter, open-label, phase 3 study (HAVEN 2)

Efficacy, safety and pharmacokinetics of once-weekly prophylactic emicizumab (ACE910) in pediatric persons (<12 years) with hemophilia A with inhibitors: interim analysis of single-arm, multicenter, open-label, phase 3 study (HAVEN 2)

AWARDED/PRESENTED: 2017
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Guy Young, Johannes Oldenburg, Ri Liesner, Victor Jiménez-Yuste, Maria Elisa Mancuso, Tiffany Chang, Marianne Uguen, Christophe Dhalluin, Christophe Schmitt, Sabine Fuerst-Recktenwald, Midori Shima, Rebecca Kruse-Jarres
Update on a phase 1/2 open-label trial of BAX 335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for hemophilia B

Update on a phase 1/2 open-label trial of BAX 335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for hemophilia B

AWARDED/PRESENTED: 2015
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Paul E. Monahan, Christopher E. Walsh, Barbara A. Konkle, Jerry S. Powell, Neil C. Josephson, Miguel A. Escobar, Scott W. McPhee, Boyan Litchev, Michael Cecerle, Bruce Ewenstein, Hanspeter Rottensteiner, Maurus De la Rosa, Birgit M. Reipert, R. Jude Samulski, Anne Prener, Friedrich Scheiflinger