Tiffany Laurel House

I never wanted to talk about Tiffany in the past tense. Never in my lifetime did I want to imagine a life without Tiffany in it or even occur to me for that matter! Tiffany was a force to be reckoned with. I met Tiffany and her family when I was 8 years old in San Antonio at my first-ever Pompe conference.

Tiffany was someone that was easy to look up to, because despite her own personal struggles living with Pompe, she always handled it with poise and grace. She always thought of others before her own challenges.

Tiffany’s life mission was to advocate for people living with this disease and bringing awareness to others for future treatment. She worked hard for years to have newborn screening (NBS) at birth so if needed, people can start treatment as soon as possible.

Tiffany knew no strangers. She had an ability to make anyone comfortable around her like they had been lifetime friends. People trusted her and everyone spoke highly of her.

She was a beacon of grace, poise, and determination until the very end. You never knew what was going on in her life because she was always first to lend a hand. She was selfless and irreplaceable.

Tiffany has left a lasting impact on my life. From the moment I met her, I was memorized by her and still am. I will forever have her wise words in the back of my mind, so even though Tiffany is no longer with us, I will hear her advice for the rest of my life, and for that, I will be eternally grateful.

– Morgan Burroughs

I first met Tiffany in 2001, when I was her research nurse during a Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More clinical trialA research study that tests new treatments or approaches in people. More. In 2003, I began working for the AMDA as the Patient AdvocateA person who helps patients and families navigate care, information, services, and support. More, and over the years my connection with Tiffany grew far beyond our shared work in the Pompe community. For 24 years, I had the privilege of knowing her not only through the AMDA, but also as a dear friend.

Tiffany was funny, warm, and full of joy. She loved her family deeply, adored her dogs, and cared for her friends in a way that made each person feel seen, welcomed, and valued.

One of the things I will always remember most about Tiffany was her generosity. She opened her home to me, and to other friends as well, when we did not have anywhere else to go on Thanksgiving or Christmas. That was simply who she was. She had a gift for making people feel like they belonged. She made difficult times easier with her kindness, her laughter, and her open heart.

Tiffany brought warmth and laughter wherever she went. Some of the happiest moments were the times spent together sharing stories, laughter, and friendship.

Tiffany gave so much of herself to the Pompe community through her work with the AMDA. She cared deeply about patients and families and worked tirelessly to make sure they felt supported, informed, and less alone. Her compassion, dedication, and vision helped shape the AMDA in meaningful ways, and her influence continues to be felt in the work of the organization today. She was not only a leader, but also a steady and caring presence for so many of us.

I feel very grateful to have known Tiffany for 24 years and to have shared so many memories with her. She touched countless lives simply by being who she was, generous, loving, funny, and real.

Tiffany is deeply missed and will always be remembered with love, gratitude, and affection.

– Marsha Zimmerman

Tiffany served on the IPA Board from 2011 to 2014, after which she was appointed Chair — a role she held until her passing. Under her leadership:

• Founded the IPA Community Advisory Board (CAB) in 2019, a pioneering, patient-led initiative now with ~30 members across three continents.
• Established International Pompe Day on April 15, with the rallying message “Together We Are Strong.” The 12th annual celebration took place in 2025.
• Co-authored a landmark peer-reviewed paper, “The Role of Patient Advocacy Organizations in Shaping Medical Research: The Pompe Model,” Annals of Translational Medicine (2019).
• Expanded the IPA-Erasmus MC Pompe Survey to reflect the global diversity of the Pompe community.
• Led the Global Pompe Data Collaboration Initiative to unify fragmented patient data across countries and research centers.
• Expanded the Pompe Connections multilingual resource library to more than 12 languages, covering respiratory health, nutrition, gene therapyA developing treatment that aims to fix, replace, or add genetic instructions. More, family planning, and emotional wellness.
• Launched the IPA Pompe Empowerment Program in the Netherlands (2015) as lead trainer, cultivating the next generation of patient advocatesA person who helps patients and families navigate care, information, services, and support. More.
• The IPA now unites representatives from over 40 countries, including Japan, India, Brazil, South Africa, across Europe, and the United States.

Tiffany was a voting patient representative on four FDA Advisory Committees and a tireless voice for patient-centered policy:

• FDA Voting Member: LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More (Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More) Advisory Committee, where she evaluated the application and provided recommendations.
• FDA Patient Representative: Niemann-Pick Type C and NAGS Deficiency Advisory Committee.
• Panelist: Patient-Focused Drug Development Meeting on Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.
• Active/Voting Member: Lysosomal Disease Network (LDN) External Advisory Committee, NIH Rare Diseases Clinical Research Network.
• Member: Lysosomal Storage Disorders (LSDA group of diseases in which substances build up inside lysosomes because the body cannot break them... More) Advocacy Coalition, focused on Medicare home infusionA method of delivering medication through an IV. More, newborn screening, and federal policy advocacy.
• Member: American College of Medical Genetics and Genomics (ACMG) Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Evidence-Based Guideline Working Group.
• Member: Pompe Registry International Board of Advisors / Octopus Project, working to standardize data collection across global patient registries.
• Advocate: Late-Onset Neuromuscular Disease Consortium (LONDC), focused on speeding referrals for patients with late-onset neuromuscular diseases.
• Contributed to more than 20 working groups, coalitions, consortia, and community advisory boards.

Peer-Reviewed Publications:

• House T, O’Donnell K, Saich R, Di Pietro F, Broekgaarden R, Muir A, Schaller T. “The Role of Patient Advocacy Organizations in Shaping Medical Research: The Pompe Model.” Annals of Translational Medicine 2019;7(13):293. DOI: 10.20137/atm.2019.04.28
  Full text here.
• Theunissen MTM, van den Elsen RM, House TL, et al. “The Impact of COVID-19 Infection, the Pandemic and Its Associated Control Measures on Patients with Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.” Journal of Neurology 271,32-45 (2024). DOI: 10.1007/s00415-023-11999-2
  Full text here.

Conference Abstract:

• “Patient Voices on E-Health.” 4th Congress in E-Health & Innovation to Overcome Barriers in Neuromuscular Disease (eNMD), Munich Symposium, November 2023.

Media:

• Featured on Mystery Diagnosis, bringing Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More to national television audiences.
• Featured in People magazine, raising public awareness about the challenges faced by rare disease patients and families.

“Even a few days’ difference in the initiation of treatment can have a profound impact on the path that these children’s lives will take. — Tiffany House”

Tiffany served on the Texas Newborn Screening Advisory Committee and spent a decade fighting to ensure that babies born with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More could be identified and treated immediately at birth. Her efforts, alongside the EverLife Foundation, Texas Rare Alliance, and over 60 advocacy groups, led to the passage of House Bill 2478 in 2023 — which passed 30-1 in the Texas Senate and was signed by Governor Greg Abbott. On August 18, 2025, Texas launched newborn screening for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. Tiffany did not live to see that day, but it is her victory.