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Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice

Wang X, Fu RY, Li C, Chen CY, Firrman J, Konkle BA, Zhang J, Li L, Xiao W, Poncz M, Miao CH

Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH

Konkle B, Pierce G, Coffin D, Naccache M, Clark RC, George L, Iorio A, O'Mahony B, Pipe S, Skinner M, Watson C, Peyvandi F, Mahlangu J

Patient-relevant health outcomes for hemophilia care: Development of an international standard outcomes set

van Balen EC, O'Mahony B, Cnossen MH, Dolan G, Blanchette VS, Fischer K, Gue D, O'Hara J, Iorio A, Jackson S, Konkle BA, Nugent DJ, Coffin D, Skinner MW, Smit C, Srivastava A, van Eenennaam F, van der Bom JG, Gouw SC.

The spectrum and severity of bleeding in adolescents with low von Willebrand factor–associated heavy menstrual bleeding

Srivaths L, Minard CG, O'Brien SH, Wheeler AP, Mullins E, Sharma M, Sidonio R, Jain S, Zia A, Ragni MV, Kulkarni R, Dietrich JE, Kouides PA.

von Willebrand Disease: Proposing definitions for future research

Connell NT, James PD, Brignardello-Petersen R, Abdul-Kadir R, Ameer B, Arapshian A, Couper S, Di Paola J, Eikenboom J, Giraud N, Grow JM, Haberichter S, Jacobs-Pratt V, Konkle BA, Kouides P, Laffan M, Lavin M, Leebeek FWG, McLintock C, McRae S, Montgomery R, O'Brien SH, O'Donnell JS, Ozelo MC, Scappe N, Sidonio R, Tosetto A, Weyand AC, Kalot MA, Husainat N, Mustafa RA, Flood VH.

Plasma factor IX: The tip of the iceberg?

Sidonio RF Jr, Batsuli G.