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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

Plasma factor IX: The tip of the iceberg?

Sidonio RF Jr, Batsuli G.

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.

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

Characterizing Female Patients with Haemophilia A: Administrative Claims Analysis and Medical Chart Review.

Farej R, Batt K, Afonja O, Martin C, Aubert R, Carlyle M, White J, Sidonio RF, Jr.