Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities for ultra-rare inherited bleeding disorders

Author:

Nugent Diane12,Acharya Suchitra S.3,Baumann Kimberly J.4,Bedrosian Camille5,Bialas Rebecca6,Brown Kai7,Corzo Deya8,Haidar Amar9,Hayward Catherine P. M.1011ORCID,Marks Peter12,Menegatti Marzia13ORCID,Miller Margaret E.14,Nammacher Kate7,Palla Roberta1315ORCID,Peltier Skye4ORCID,Pruthi Rajiv K.16ORCID,Recht Michael1718ORCID,Sørensen Benny19,Tarantino Michael20,Wolberg Alisa S.2122ORCID,Shapiro Amy D.23ORCID

Affiliation:

1. Center for Inherited Blood Disorders, Orange, California, USA

2. Pediatric Hematology Division, Department of Pediatrics, University of California at Irvine, Children's Hospital of Orange County, Irvine, California, USA

3. Hemostasis and Thrombosis Center, Northwell Health, New Hyde Park, New York, New York, USA

4. Center for Bleeding and Clotting Disorders, M Health Fairview, Minneapolis, Minnesota, USA

5. Ultragenyx Pharmaceutical Inc Novato, California, USA

6. Plasminogen Deficiency Foundation, Durham, North Carolina, USA

7. National Hemophilia Foundation, New York, New York, USA

8. Sigilon Therapeutics, Cambridge, Massachusetts, USA

9. Patient author, Lived Experience Expert, Dearborn, Michigan, USA

10. Hamilton Regional Laboratory Medicine Program, Hamilton Health Sciences, Hamilton, Ontario, Canada

11. Department of Pathology and Molecular Medicine, McMaster University, Hamilton, Ontario, Canada

12. Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA

13. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy

14. Oncology Support Service, Providence Alaska Medical Center, Anchorage, Alaska, USA

15. Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy

16. Comprehensive Hemophilia Center, Division of Hematology, Mayo Clinic, Rochester, Minnesota, USA

17. American Thrombosis and Hemostasis Network, Rochester, New York, USA

18. The Hemophilia Center, Oregon Health & Science University, Portland, Oregon, USA

19. Hemab Therapeutics, Copenhagen, Denmark

20. Bleeding and Clotting Disorders Institute, Peoria, Illinois, USA

21. Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA

22. UNC Blood Research Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA

23. Indiana Hemophilia and Thrombosis Center, Indianapolis, Indiana, USA

Funder

Summit and this manuscript were funded by National Hemophilia Foundation

Publisher

Informa UK Limited

Subject

Hematology

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