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Inherited Platelet Disorders: An Updated Overview

Palma-Barqueros V, Revilla N, Sánchez A, Zamora Cánovas A, Rodriguez-Alén A, Marín-Quílez A, González-Porras JR, Vicente V, Lozano ML, Bastida JM, Rivera J.

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.