Journal article
Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders
Abstract
BACKGROUND: Ultra-rare inherited bleeding disorders (BDs) present important challenges for generating a strong evidence foundation for optimal diagnosis and management. Without disorder-appropriate treatment, affected individuals potentially face life-threatening bleeding, delayed diagnosis, suboptimal management of invasive procedures, psychosocial distress, pain, and decreased quality-of-life.
RESEARCH DESIGN AND METHODS: The National …
Authors
Nugent D; Acharya SS; Baumann KJ; Bedrosian C; Bialas R; Brown K; Corzo D; Haidar A; Hayward CPM; Marks P
Journal
Expert Review of Hematology, Vol. 16, No. sup1, pp. 55–70
Publisher
Taylor & Francis
Publication Date
March 15, 2023
DOI
10.1080/17474086.2023.2175661
ISSN
1747-4086