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

On My Own, A Pilot Transition Program for Teens

On My Own, A Pilot Transition Program for Teens

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Collaboration/Team Models
RESEARCHERS:
Amanda Fridley, Pharm D, Cincinnati Children's Hospital Medical Center; Anna Worpenberg, MSW, LSW, Cincinnati Children's Hospital Medical Center; Helen Lamping, Associates, Tri-State Bleeding Disorder Foundation; Julie Doyle, BSN, RN, Cincinnati Children's Hospital Medical Center; Julie Hendrickson, BSN, RN, CPN, Cincinnati Children's Hospital Medical Center; Lisa Littner, MPH, MSW, LISW-S, CHES, Cincinnati Children's Hospital Medical Center; Mary Busam, Pharm D, Cincinnati Children's Hospital Medical Center; Molly Mays, MPT, Cincinnati Children's Hospital Medical Center; Shanna Korn, BA, Cincinnati Children's Hospital Medical Center
Characterization of the Early Immune Response to Factor VIII

Characterization of the Early Immune Response to Factor VIII

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
RESEARCHERS:
Glaivy Batsuli, MD, Emory University; Wallace Baldwin, MS, Emory University; Jasmine Ito, BS, Emory University; John Healey, BS, Emory University; Shannon Meeks, MD, Emory University; Seema Patel, PhD, Emory University
Overview of the clinical development of fitusiran

Overview of the clinical development of fitusiran

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
RESEARCHERS:
Baisong Mei, MD, PhD, Sanofi; Naghmana Bajwa, MD, Sanofi; Fadi Shammas, MD, Sanofi; Salim Kichou, MD, Sanofi; Shauna Andersson, M.D., PhD, Sanofi
A Novel Approach for Rare Bleeding Disorders: Shielded Living Therapeutics

A Novel Approach for Rare Bleeding Disorders: Shielded Living Therapeutics

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
RESEARCHERS:
Christine Carroll, BS, Sigilon Therapeutics; David Peritt, PhD, Sigilon Therapeutics; Devyn Smith, PhD, Sigilon Therapeutics; Deya Corzo, MD, Sigilon Therapeutics; Guillaume Carmona, PhD, Sigilon Therapeutics; Jared Sewell, PhD, Sigilon Therapeutics; Janet Huang, MS, Sigilon Therapeutics; Kevin Lai, PhD, Sigilon Therapeutics; Lauren Barney, PhD, Sigilon Therapeutics; Michaela Toland, BS, Sigilon Therapeutics; Michele McAuliffe, BS, Sigilon Therapeutics; Susan Drapeau, PhD, Sigilon Therapeutics; Rogerio Vivaldi, MD, MBA, Sigilon Therapeutics; Verna Zhao, BS, Sigilon Therapeutics
Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after Von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database

Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after Von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Abioloa Oladapo, PhD, Takeda Pharmaceuticals, USA; Imrran Halari, MS, Charles River Associates; Jonathan Roberts, MD, Bleeding and Clotting Disorders Institute; Lynn Malec, MD, MSc, Blood Research Institute, Versiti; Robert Sidonio, MD, Emory University and Children's Healthcare of Atlanta; Sarah Hale, PhD, Takeda Pharmaceuticals, USA
Development of the WFH-ISTH Gene Therapy Registry

Development of the WFH-ISTH Gene Therapy Registry

AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
RESEARCHERS:
Barbara Konkle, MD, Bloodworks NW, Washington Center for Bleeding Disorders; Brian O’Mahony, NA, Irish Haemophilia Society; Cary Clark, NA, International Society on Thrombosis and Haemostasis Inc; Crystal Watson, NA, American Thrombosis and Hemostasis Network; Donna Coffin, MSc, World Federation of Hemophilia; Flora Peyvandi, MD, PhD, IRCCS Maggiore Hospital Milan and University of Milan; Lindsey A. George, MD, The Children's Hospital of Philadelphia; Glenn F. Pierce, MD, PhD, World Federation of Hemophilia; Alfonso Iorio, MD, McMaster University; Johnny Mahlangu, MD, Haemophilia Comprehensive Care Centre, University of the Witwatersrand, NHLS; Mayss Naccache, MSc, World Federation of Hemophilia; Mark W. Skinner, JD, Institute for Policy Advancement Ltd.; Steven W. Pipe, MD, University of Michigan, Pediatrics