Looking forward to greater progress in our community in 2024

Embracing the start of 2024, I reflect on the friendships I’ve made within the hemophilia community over the past year. As a woman navigating the complexities of hemophilia B and von Willebrand disease, these connections have become a source of strength and solidarity for me. Each person I’ve encountered embodies resilience, and collectively we stand as a testament to the unwavering spirit that defines our community. Proud of the bonds formed, I eagerly anticipate the growth of these newfound friendships in 2024.

Engaging in conversations with people holding diverse perspectives has been truly enlightening. Caregivers, mothers, daughters, and fathers all have perspectives and experiences different from mine. In particular, discussions with mothers have underscored the remarkable progress achieved in treating severe hemophilia over the past few years — a stride that can only be described as revolutionary.

For example, Hemlibra (emicizumab-kxwh) has been a beacon of progress for those grappling with hemophilia A. This groundbreaking product has transformed the treatment narrative, offering a departure from the traditional intravenous infusions and the advent of a simpler and more manageable injection method. As we bid farewell to the challenges of the past, the promise of Hemlibra heralds a future where families can navigate the complexities of hemophilia with greater ease and normalcy.

Simultaneously, those of us with hemophilia B find hope in the form of Hemgenix (etranacogene dezaparvovec), a gene therapy drug that whispers promises of lasting relief. CNN called it the most expensive drug in the world, at least in 2022. The price tag puts it out of reach for most.

Acknowledging challenges

Yet even as we celebrate these victories, a sobering reality persists. Many within the hemophilia community, especially women, those in less developed countries, and those with milder forms of the condition, find themselves on the fringes of progress. In addition, the gap between treatment options for hemophilia and von Willebrand disease remains a poignant reminder that our journey toward inclusivity and comprehensive care is still in its infancy.

As we enter the new year, let’s celebrate the scientific triumphs and acknowledge the tenacity of individuals and families grappling with hemophilia. Their stories of resilience, adaptability, and unwavering hope are the heartbeat of the community. Each injection, each milestone, and each triumph against the odds is a testament to the strength that unites us.

In the spirit of the new year, let us look forward with hope. Hope for further breakthroughs, hope for greater inclusivity, and hope for a world where the challenges of hemophilia are met with compassion, understanding, and a relentless pursuit of solutions.

May 2024 be the year when the promise of “treatment for all” is not just a distant dream, but a tangible reality.