A Legacy of Leadership, Love, and Lifesaving Work in the Pompe Community
This August, the Acid Maltase Deficiency Association (AMDA) turns 30. Thirty years of progress. Thirty years of advocacy. Thirty years of showing up for patients and families around the world.
When Randall and Marylyn House founded the AMDA in August 1995, they had one powerful mission in mind: to generate support for the scientific community so that effective treatments—first enzyme therapy, then gene therapy—could become a reality for those living with Pompe disease.
At the time, there was no treatment, no community infrastructure, and no roadmap. But what they started would spark a movement.
The Early Years: Science First
In 1998, just three years after the AMDA’s founding, the organization co-hosted a conference with the National Institutes of Health, building momentum and trust with the research community. A year later, the first ERT trials for Pompe began—in the Netherlands and at Duke University.
Then in 2001, the AMDA co-hosted its first International Scientific Research and Patient Conference with the International Pompe Association (IPA). For the first time, patients heard the results of clinical trials directly from the scientists themselves. It was historic—and a sign of things to come.
A Shift Toward the People
By 2003, the AMDA had grown into more than a research support group. With the addition of Marsha Zimmerman as Patient Advocate, the organization took a major step toward becoming what it is today; a hybrid force for both scientific progress and community connection.
That same year, the AMDA began hosting teleconferences—a major leap forward in accessibility. These one-hour virtual sessions gave families across the world a chance to hear directly from researchers several times a year, instead of waiting four or five years for in-person conferences. It was a game-changer.
In 2010, the AMDA elevated this model again by launching webinars—adding visual presentations and data, enhancing understanding, and creating deeper engagement.
More recently, in response to mental health workshops held during the 2024 hybrid conference, the AMDA launched the “Talking With Your Pompe Peeps” series—a community-centered digital forum moderated by patients and caregivers. Focused on real conversations about the lived Pompe experience, the series marks a new phase in our shift toward empowering the community to lead the discussions most important to them.
Milestones That Mattered
- April 2006: The FDA approved Myozyme, the first-ever commercial treatment for Pompe.
- November 2006: The AMDA hosted its second International Conference, bringing even more visibility and unity to the Pompe community.
- 2011: The first “Pull for Pompe” fundraiser was launched in partnership with PCMA (Precast Concrete Manufacturers Association), raising funds to directly support research and patient programs
- 2013: The AMDA launched the Helen Walker Research Grant, ensuring that 100% of awarded funds go directly to Pompe disease research.
- 2015: After years of advocacy, Pompe disease was officially added to the U.S. Recommended Uniform Screening Panel (RUSP) for newborn screening—a victory that would save lives.
- 2023: The AMDA contributed to the Texas Advisory Committee’s support of House Bill 2478, setting a statewide deadline for Pompe to be added to the newborn screening panel.
The People Behind the Progress
Over the years, the AMDA continued to grow—one milestone at a time. Each new addition to the team was a turning point.
- 2011: Tiffany House, the very person this organization was built for, stepped into the role of President.
- 2018: Morgan Burroughs joined as Social Media Coordinator, expanding the AMDA’s online reach.
- 2021: Juls Williams became the AMDA’s Liaison, helping streamline operations and communication.
- 2022: Matt Zimmerman came on as Director of Communications/IT, bringing new digital life and infrastructure to the AMDA.
- 2024: Andrea Faris joined as Lead Social Media Manager, shifting the organization’s social media approach from news sharing to strategic community engagement.
- 2025: Tara Mancine steps in as Lead Social Media Manager, and Andrea Faris became Director of the AMDA, carrying the vision forward.
Three decades of impact. Here’s a look at some of the major milestones that shaped the AMDA’s journey:
Tiffany’s Legacy
But no person shaped the AMDA more than Tiffany House.
She was the face, the force, and the heart of this organization. Tiffany wasn’t just a figurehead—she was the reason people believed in what the AMDA could be. She gave everything she had, quietly and without ever asking for praise. Whether fighting for better treatment access or ensuring patients had a seat at the table, Tiffany did it all with one thing in mind: the Pompe community.
When she passed away May 25, 2025, the loss was—and still is—immeasurable.
Carrying the Dream Forward
As we mark this 30-year milestone, it’s impossible not to feel the absence of Tiffany’s voice. But what she built—what we all built together—continues.
We remain as committed as ever to supporting scientific discovery, connecting patients and families, and building a better future for everyone living with Pompe disease.
This isn’t just a birthday.
It’s a promise.
To Tiffany.
To the families.
To the science.
To the future.
We will continue the work she dedicated her life to.