Visionary and Kind Leader, Tireless Advocate, Beloved Champion of the Pompe Community, True Friend
It is with deep sorrow and profound respect that we remember Tiffany House, whose life and legacy continue to inspire all who were fortunate enough to know her. Tiffany was not only a trailblazer in the fight against Pompe disease—she was the heart of a global movement.
Diagnosed at just 12 years old, Tiffany defied the odds placed before her. What some called a grim prognosis, she transformed into a life of purpose, courage, and extraordinary impact. When doctors told her she would not live past 20, she helped change the science that would prove them wrong.
Tiffany was one the first Pompe patients in the world to receive enzyme replacement therapy. Her courageous participation in the groundbreaking clinical trial led by Dr. Ans van der Ploeg was not only brave—it was transformative. Thanks to their relentless dedication over more than a decade, the first successful treatment for Acid Maltase Deficiency (Pompe disease) was developed. Tiffany often said she owed her life to her parents, but she also credited the entire “Pompe Team” in Holland, who gave not only medicine but also compassion and humanity—something she carried in her heart always.
Tiffany’s journey was never one she traveled alone. Her devoted parents, Marylyn and Randall House, were foundational in building the Pompe community. Randall, a co-founder of the International Pompe Association (IPA), helped lay the groundwork for a truly united global Pompe network. While Tiffany was not present at the IPA’s founding, she later became deeply involved as Randall went on to serve as Chair of the organization. Together, the House family became an unstoppable force for progress, advocacy, and patient-centered care—building lasting bridges between families, clinicians, and researchers so no Pompe patient would ever feel isolated or unheard.
It is extraordinarily rare for a rare disease to have even one treatment—let alone multiple options. As members of the rare disease community, we recognize how uniquely fortunate the Pompe community is in this regard, largely thanks to the House family’s tireless efforts. Yet, Tiffany always reminded us that despite this progress, the work is far from over.
Tiffany was a force. A driving energy connecting patients, scientists, clinicians, and industry—always pushing for better care, stronger research, and true collaboration. This inspired what is now known as the Pompe Model—a powerful and enduring framework uniting the patient community, the medical/scientific community, and industry around a shared mission. Tiffany didn’t just champion this model—she helped build it.
In recognition of this groundbreaking work, Tiffany and the IPA co-authored a landmark article titled “The role of patient advocacy organizations in shaping medical research: the Pompe model,” which was accepted for publication in 2019 in the Annals of Translational Medicine, an international, peer-reviewed journal. The article explores the Pompe Model’s two-decade legacy of patient-driven progress, collaborative research, and shared achievement. As a united community of patients, families, friends, physicians, scientists, and industry partners, we have shown—thanks in large part to Tiffany’s leadership—that when we work together, we are undeniably stronger and capable of making lasting, life-saving change.
Among her countless contributions, Tiffany had great ideas—and when she had them, she made them real. For example, she was instrumental in creating the IPA’s Community Advisory Board (CAB) in 2019, a patient-led initiative uniquely organized and operated by the Pompe patient community rather than being industry-driven. Traditionally, a CAB is a neutral group formed by patient advocates to advise on the latest developments in medical research and to facilitate dialogue with research sponsors. The IPA CAB now includes about 30 members from multiple countries across three continents, representing a broad spectrum of Pompe perspectives. It is an extremely valued resource by industry seeking expert patient feedback.
Tiffany was also the driving force behind the creation of International Pompe Day. Thanks to her vision and leadership, the IPA established April 15 as a global day of awareness, united under the powerful slogan: “Together We Are Strong.” April 15, 2025 marked the 12th Annual International Pompe Day—a lasting tribute to the community she helped build and the impact she made. Through this initiative and so many others, Tiffany effortlessly fostered meaningful relationships among patients, families, clinicians, scientists, and industry—always working to deepen understanding and address the unmet needs of the Pompe community.
She was a relentless advocate for important international campaigns and initiatives dear to her heart, including, but not limited to:
Dose Flexibility: Tiffany House was a steadfast advocate for this cause-championing the principle that every patient deserves access to the most effective treatment possible. She tirelessly pushed for solutions that would allow for dose optimization based on patient need, not economic constraints, ensuring that this issue remains a global priority.
Treatment Transition and Expectation Guidelines: Tiffany understood the critical need for patients to have realistic expectations about treatment response, especially as new therapies emerge. She worked tirelessly to provide guidance on treatment choices and transitions, emphasizing the importance of informed decision-making in collaboration with physicians.
Advocacy for Patient Reported Outcomes and Clinical Trial Design: Tiffany House was a passionate advocate for smarter, more inclusive clinical research that truly reflected the lived experiences of Pompe patients. She understood that one of the greatest challenges facing future therapies is how to design meaningful trials for an ultra-rare disease with a heterogeneous, late-onset population. Under her leadership, the IPA consistently championed the importance of placing greater value on Patient-Reported Outcomes (PROs) – not only in trial design but also in regulatory review. Tiffany believed deeply that with small trial sizes and wide variability in disease progression and treatment response, the patient voice must be central. She pushed for the broadest possible evaluation criteria to ensure all potential benefits of treatment, including symptom stabilization, are recognized and valued. Her vision continues to shape a more patient-centered future in Pompe research.
IPA-Erasmus MC Pompe Survey: Launched in 2002 as a collaboration between the IPA and Erasmus MC, this survey has been a cornerstone for capturing patient-reported outcomes and natural disease history. Under Tiffany’s guidance, the survey was expanded and updated with new languages and modules, ensuring the Pompe community’s voice remains central to research.
Global Pompe Data Collaboration: Recognizing the fragmentation of Pompe patient data worldwide, Tiffany helped spearhead efforts to unify data collection and sharing across registries and research centers. This initiative aims to break down silos, accelerate research, and provide patients with meaningful insights into their disease and treatments.
The Pompe Connections, Empowering Patients Through Knowledge: Recognizing the need for accurate, accessible, and multilingual information, Tiffany House championed The Pompe Connections campaign, an ongoing initiative to equip Pompe patients and families with medically reviewed resources to support daily life and long-term care. Available in over a dozen languages, including Arabic, Chinese, Dutch, French, German, Italian, Japanese, Korean, Portuguese, Russian, Spanish, and more, The Pompe Connections and its Treatment Edition cover essential topics such as respiratory health, nutrition, exercise, physical therapy, emotional well-being, and family planning. This campaign reflects Tiffany’s unwavering belief that informed patients are empowered patients, and it continues to serve as a vital tool for the global Pompe community.
Newborn Screening (NBS) Advocacy: Tiffany was a passionate advocate for expanding newborn screening for Pompe disease, knowing early diagnosis can prevent irreversible muscle damage by enabling timely treatment. She championed not only the implementation of NBS but also the development of tailored support for families, especially those with late-onset diagnoses who face uncertainty about when symptoms may appear. Tiffany’s leadership helped ensure the community’s needs are heard and met at every stage of Pompe disease.
AMDA/IPA International Pompe Patient and Scientific Conference: First organized in 1996 by the Acid Maltase Deficiency Association (AMDA), this landmark conference was the first to bring together scientists, clinicians, and patients involved in Pompe research from around the globe. Held every four years, and later co-hosted by the International Pompe Association (IPA), the conference created a transformative space where scientific collaboration and patient engagement could thrive. It fostered a sense of community among researchers and helped shift the dynamic between scientists and patients toward a more collaborative and human-centered model. Tiffany House championed this spirit of unity and dialogue, recognizing that scientific progress and patient experience must move forward hand in hand.
IPA Pompe Empowerment Program (2015, Netherlands): In 2015, the IPA launched the Pompe Empowerment Program in the Netherlands—an initiative designed to train and inspire the next generation of Pompe patient advocates. Tiffany House served as one of the lead trainers, sharing her experience, wisdom, and advocacy skills with emerging patient leaders from around the world. Through mentorship and hands-on learning, the program equipped participants to take on active roles in shaping the future of Pompe advocacy. Tiffany’s guidance left a lasting impression, helping to cultivate a new wave of empowered voices in the community.
As President of the Acid Maltase Deficiency Association (AMDA) and Chair of the IPA, Tiffany championed the inclusion of patient voices at every table. She didn’t just ask to be heard—she made sure the entire community had a voice in shaping the future of Pompe research and treatment.
Her work extended far beyond advocacy. She answered late-night emails, explained complex research in compassionate terms, held the hands of newly diagnosed families, and shared in the triumphs and heartbreaks of an entire community. Her empathy knew no borders.
Tiffany lived with grace and strength in the face of unrelenting challenges. She never stopped fighting—not for herself, and never just for herself. She carried all of us with her.
Her legacy lives on in every patient who now has access to treatment, in every family that finds hope where there once was none, and in every step forward the Pompe community continues to take. She showed us what true advocacy looks like: bold, relentless, compassionate, and kind.
We extend our deepest condolences to Tiffany’s family, friends, and the countless people whose lives she touched. We mourn her loss, but more than anything—we honor her life.
Thank you, Tiffany, for your passion. Thank you for your voice, your courage, and your heart.
You will forever remain a beacon of hope, a symbol of strength, and a cherished friend to us all.
International Pompe Association