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

Characterization of the Early Immune Response to Factor VIII
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research

Overview of the clinical development of fitusiran
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

A Novel Approach for Rare Bleeding Disorders: Shielded Living Therapeutics
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Biomedical/Coagulation Research

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

Development of the WFH-ISTH Gene Therapy Registry
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Real-world treatment patterns, health outcomes, and healthcare resource use among persons with hemophilia A
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

The World Federation of Hemophilia World Bleeding Disorders Registry: A Two-year Update
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Progress Update on the Development of Etranacogene Dezaparvovec (AMT-061) in Severe or Moderately Severe Hemophilia B
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Anti-Gravity Treadmill Use in an Adult with Severe Hemophilic Arthropathy: A Case Report.
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Orthopedic and Physical Therapy

An analysis of fatalities in persons with congenital hemophilia A (PwcHA) reported in the FDA Adverse Event Reporting System (FAERS) database
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Evaluating BIVV001, a New Class of Factor VIII Replacement Therapy: A Phase 3 Study (XTEND-1) Design
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Characteristics of persons with hemophilia A treated with emicizumab with or without factor VIII inhibitors
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Non-severe hemophilia is not benign? - Insights from the PROBE Study
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Redefining Treatment Satisfaction and Its Impact on Treatment Adherence and Value for Persons with Hemophilia: Findings from the HemACTIVE Study
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Fear of pain in people with hemophilia and their families – a pilot study
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Psychosocial Issues

The Moti-VIII Study – Factors for Empowering Mobility and Well-being in Hemophilia A
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

A US payer database algorithm to identify clinical profiles of hemophilia B for burden of illness assessment
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

Behavior and cognition in children and young adults with hemophilia A or B: an update on developmental outcome
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Clinical Research/Clinical Trials

Incidence and Prevalence of Diagnosed and Undiagnosed Hemophilia A and Hemophilia B in the USA
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research

A Review of Current Patient Reported Outcome Measures Used to Assess Mental Health in People with Hemophilia
AWARDED/PRESENTED: 2020
GRANT/PROGRAM:
Bleeding Disorders Conference
Quality of Life/Outcomes Research