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
Objectives: Von Willebrand disease (VWD) is the most common bleeding disorder in children and adolescents. Its varied clinical presentation contributes to challenges and delays in diagnosis and management. We characterized…
Supporting patient voice to inform healthcare decision-making: a discrete choice experiment on disability paradox in hemophilia
Case Report of Surgical Management of a Hemophilia B Gene Transfer Clinical Trial Participant Following Etranacogene Dezaparvovec (AMT-061) Gene Therapy
Real-world treatment patterns, health outcomes, and healthcare resource use among persons with hemophilia A
Objective: Understanding characteristics of the aging hemophilia A (HA) population is critical to informing appropriate management strategies. There is paucity of published literature describing the aging HA population. This…
An ECHO’d Practice: Utilizing Tele-Mentoring for Enhanced Data Quality Across One Hemophilia Treatment Center Region
Background: In Spring 2017, the Western States/Region IX Hemophilia Treatment Center Network (WSRHN) Coordinating Committee expanded representation to Data Manager/Clinical Research Coordinators (DM/CRC) to address two …
Vector DNA clearance from bodily fluids in patients with severe or moderate-severe hemophilia B following systemic administration of AAV5-hFIX and AAV5-hFIX Padua
Progress Update on the Development of Etranacogene Dezaparvovec (AMT-061) in Severe or Moderately Severe Hemophilia B
A single administration of AAV5-hFIX in newborn, juvenile and adult mice leads to stable hFIX expression up to 18 months after dosing
A contemporary framework for understanding mortality in people with congenital hemophilia A (PwcHA)
An analysis of fatalities in persons with congenital hemophilia A (PwcHA) reported in the FDA Adverse Event Reporting System (FAERS) database