NetWorks Issue 17: The Design Issue

A Transformative Journey

Fighting Hereditary Angioedema (HAE) with Determination and Innovation

In a profound breakthrough that promises to redefine the landscape of Hereditary Angioedema (HAE) treatment, phase one clinical trials for gene therapy have yielded extraordinary results, evoking a spectrum of emotions for Ann Harding, Chair of HAE UK and Director of Quattro Road Services. HAE, a rare genetic disorder affecting 1 in 50,000 individuals, inflicts its victims with unpredictable bouts of swelling throughout the body, with the potential for life-threatening consequences, especially when the swelling encroaches upon the throat. For Ann, this scientific milestone strikes a deeply personal chord as her daughter, Sian, has endured the tumultuous journey of living with HAE since birth.

The trials had participants from across the United Kingdom, New Zealand, and the Netherlands, and the outcomes surpassed expectations, unveiling significant advancements in symptom management and a temporary cessation of debilitating attacks. Ann, overwhelmed with gratitude, acknowledges the collective efforts of medical professionals, trustees, donors, and pharmaceutical entities whose collaborative spirit propelled this groundbreaking research forward.

Dr. Padmalal Gurugama, spearheading the UK branch of research at Cambridge University Hospital, extols the transformative potential of this therapy in elevating the quality of life for individuals grappling with HAE’s relentless onslaught.

Dr. Hilary Longhurst, Senior Medical Officer at Auckland District Health Board, lauds the indispensable role played by patient organizations like HAE UK in not only facilitating treatment development but also in ensuring equitable access to these life-changing therapies.

The reverberations of this scientific breakthrough have resonated across esteemed publications such as The Times, The Guardian, and the BBC website, underscoring the global significance of the research. John Murphy, Managing Director of Quattro, reiterates his company’s steadfast commitment to supporting HAE UK, reaffirming their dedication to seeing the research through to fruition and making tangible impacts in the lives of those affected by HAE.

Amidst the backdrop of scientific triumph, Ann Harding’s appointment as Chairman of the Hereditary Angioedema Charity emerges as a poignant testament to her indomitable spirit and unwavering advocacy on behalf of the HAE community. Her journey, intricately interwoven with Sian’s, epitomizes the resilience and fortitude required to navigate life’s most formidable challenges.

Sian’s firsthand encounters with HAE unveil the stark realities of the condition, underscoring the imperative of heightened awareness and advocacy for improved treatment modalities. Her unwavering resolve, buoyed by Ann’s steadfast support, embodies the power of familial bonds in surmounting adversity and instilling hope in the face of uncertainty.

As they traverse the intricate landscape of HAE, Ann and Sian remain steadfast in their commitment to championing the cause of those affected by this debilitating condition. Their unwavering resolve, coupled with a fervent dedication to driving positive change, serves as a beacon of hope for the HAE community and inspires others to confront life’s adversities with resilience and unwavering determination.

Through their collective efforts, Ann and Sian symbolize the transformative potential of unwavering advocacy and innovation in reshaping the narrative surrounding rare genetic disorders like HAE. Their journey epitomizes the triumph of the human spirit and underscores the profound impact of perseverance, compassion, and collective action in effecting meaningful change on a global scale.

For advice or support about HAE, please visit: https://www.haeuk.org/