Leonard A Valentino

Abstract As more advanced cell and gene therapies, including gene editing technologies, progress through drug development, there is increased emphasis on the importance of stakeholders, including people living with disease, caregivers, and healthcare professionals, to communicate using clear, accurate, and consistent language.

1 Commentary The shared decision-making (SDM) tool developed by the World Federation of Hemophilia (WFH) and described by Coffin et al. [1] provides a clear and concise overview of the process by which a person with haemophilia (PwH) can collaborate with their healthcare professionals (HCPs) to engage and arrive at a therapeutic decision [2].

1 INTRODUCTION Haemophilia treatment has progressed rapidly in the last few decades, leading to the wide availability of safe and effective factor replacement therapy.1-4 Despite such progress, many people with haemophilia (PWH) face substantial treatment burdens due to breakthrough bleeding, progressive joint disease, mental health issues, and the necessity for chronic, frequent intravenous infusions, resulting in reduced quality of life.2, 5, 6 Advances in gene editing technologies like the...