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
A Multi-System Evaluation of von Willebrand Factor Function in Type I von Willebrand Disease Mutations

A Multi-System Evaluation of von Willebrand Factor Function in Type I von Willebrand Disease Mutations

Year:
-
Grants:
Judith Graham Pool Postdoctoral Research Fellowship
Von Willebrand Disease
Author(s):
Christopher Ng

Dr. Christopher Ng was a pediatric hematology/oncology fellow at the University of Colorado - Anschutz Medical Campus. Dr. Ng attended medical school at the Keck School of Medicine at the University of Southern California and completed his pediatrics residency at the University of Washington/Seattle Children's Hospital. Dr. Ng received the NHF-Baxalta Clinical Fellowship in 2013. Dr. Ng's 2015 JGP research fellowship award project focused on a multi-system evaluation of von Willebrand factor function in Type 1 von Willebrand Disease mutations.

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia A and B with and Without Inhibitors: Interim Results from a Phase 2 Extension Study

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia A and B with and Without Inhibitors: Interim Results from a Phase 2 Extension Study

AWARDED/PRESENTED: 2017
GRANT/PROGRAM:
Bleeding Disorders Conference
New Products
RESEARCHERS:
Margaret V. Ragni, Pencho Georgiev, Tim Mant, Michael Desmond Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Savita Rangarajan, Chang-Heok Soh, Amy Monpara, Huy Van Nguyen, Kate Madigan, K John Pasi