Giving Choice a Voice: Commentary on Development of the World Federation of Hemophilia Shared Decision-Making Tool.

The shared decision-making (SDM) tool developed by the World Federation of Hemophilia (WFH) provides a clear and concise overview of the process by which people with haemophilia (PwH) can collaborate with their healthcare professionals (HCPs) to engage and arrive at a therapeutic decision. This tool will be useful for all people with all bleeding disorders, not just PwH.

One day at a time: Life with Glanzmann thrombasthenia - Qualitative results from the GT 360 study.

Introduction: Glanzmann thrombasthenia (GT) is a platelet function disorder. Symptoms include bruising and bleeding, which may be severe and life-threatening. The day-to-day experiences of those affected remain poorly documented.

Bleeding and quality of life in people with Glanzmann thrombasthenia-insights from the Glanzmann's 360 study.

Background: Glanzmann thrombasthenia (GT) is a rare platelet function disorder that results in severe bleeding. We assessed clinical symptoms and psychological parameters to identify the unmet needs associated with GT.

Objectives: Glanzmann's 360 is a mixed-methods study designed to give a contemporary snapshot of the impact of living with GT.

A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.

Introduction: Despite the progress in gene editing platforms like CRISPR/Cas9 with the potential to transform the standard of care for haemophilia, the language used to explain and discuss gene editing is not aligned across the haemophilia community. Here, we present the objective and rationale for developing a clear, consistent, and globally aligned gene editing lexicon to address these communication gaps.

Methods: Effectively communicating complex gene editing concepts requires a clear and consistent vocabulary.