Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders.
Diane NugentSuchitra S AcharyaKimberly J BaumannCamille BedrosianRebecca BialasKai BrownDeya CorzoAmar HaidarCatherine P M HaywardPeter MarksMarzia MenegattiMargaret E MillerKate NammacherRoberta PallaSkye J PeltierRajiv K PruthiMichael RechtBenny SørensenMichael TarantinoAlisa S WolbergAmy D ShapiroPublished in: Expert review of hematology (2023)
Centralization and expansion of expertise and resources, flexible innovative research and regulatory approaches, and inclusion of all people with ultra-rare inherited BDs and their health care professionals will be essential to capitalize on the opportunities outlined herein.