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Introduction
Consensus over the definition of recombinant factor VIII (rFVIII) product classification in haemophilia A is lacking. rFVIII products are often classified as standard half‐life (SHL) or extended half‐life (EHL); despite this, no universally accepted definition currently exists. One proposed definition includes half‐life, area under the curve, and technology designed to extend half‐life;...
Introduction
With recent approval of the first two gene therapies for haemophilia A and B, educational materials about AAV‐based gene therapy are needed by the haemophilia community for a better understanding of this novel therapeutic approach and helping healthcare providers and patients making personalized choices amongst an increasing array of therapeutic options.
Aim
To provide a comprehensive...