NBDF showcases a wide array of findings from the latest in bleeding disorders research at our annual Bleeding Disorders Conference.
AMT-061 (AAV5-Padua hFIX variant) an Enhanced Vector for Gene Transfer in Adults with Severe or Moderate-Severe Hemophilia B: Follow-up up to 9 Months in a Phase 2b trial
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
Quality Improvement: An Initiative to Foster Mental Health Wellness among a Hemophilia Treatment Center Patient Population
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Psychosocial Issues
Baseline patient characteristics in ReITIrate: A prospective study of rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in patients who have failed previous ITI attempts
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
Optimizing language for effective communication of gene therapy concepts: A qualitative study
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Peer Support/Outreach/Integration Models
Five-year safety and efficacy of N8-GP (ESPEROCT®) in previously treated children with hemophilia A in the completed pathfinder 5 trial
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
Quality of life and health in patients with Haemophilia in Mexico
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Four-year safety and efficacy of N8-GP (ESPEROCT®) in previously treated adolescents/adults with hemophilia A in the completed pathfinder 2 trial
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials
Patients Report High Satisfaction with US Hemophilia Treatment Centers: National Trends 2014 and 2017
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Navigating the Emergency Department: A Collaboration Among Hemophilia Treatment Center Staff, Emergency Department Staff & Bleeding Disorder Chapter Staff
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Collaboration/Team Models
Patient Perspectives on the Impact of Severe or Moderate Hemophilia on Physical Activity: HemACTIVE Survey Findings from the US and Canada
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
rFVIIIFc for first-time immune tolerance induction therapy: interim results from the global, prospective verITI-8 study
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Inhibitors
World Federation of Hemophilia Annual Global Survey 2017 – 19 years of reporting
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Validation of a FVIII Chromogenic Nijmegen Bethesda Assay for the Detection of Inhibitors in the Presence of Emicizumab (ACE-910)
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Inhibitors
The Patient Reported Outcomes Burdens and Experiences (PROBE) Study Questionnaire Development and Validation
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research
Tackling a New Era of Treatment in Hemophilia A: One Institution's Experience of Integrating Emicizumab into Practice
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
New Products
A retrospective chart review to assess clinical characteristics of women and girls with factor VIII and IX deficiency
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Women's Research
Evaluation of Joint Bleeds Using Portable Ultrasound and Its Impact on Treatment of Persons With Hemophilia in a Resource Limited Setting
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Orthopedic and Physical Therapy
A Unique Combination of Severe Congenital Factor XIII Deficiency and Type 2M Von Willebrand Disease – A Case Report
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research
Parents Empowering Parents (PEP) Community Survey: How does the community want to stay engaged, communicate, and receive information?
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Psychosocial Issues
Bleeding types and treatments in patients with von Willebrand disease before and after diagnosis
AWARDED/PRESENTED: 2019
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials