About the AMDA Research Grant
The AMDA Research Grant funds research dedicated exclusively to Pompe disease, fostering collaboration among scientists working to improve patient outcomes. Established in 2010, the program was renamed the Helen Walker Research Grant in 2013 to honor patient advocate Helen Walker, and returned to its original name in 2026 in honor of Tiffany L. House, former AMDA president and lifelong champion of the Pompe community. Made possible through the generosity of the Pompe community and initiatives like Pull for Pompe, the grant carries forward both women’s legacies of compassion and advocacy.
Research Funding Highlights
Projects Funded
Fund's Awarded
2026 - 2027 Grant Cycle
One grant of up to $150,000, for a 12- or 24-month project period.
Status: Applications Open — Letters of Intent due October 15, 2026
News & Updates
The AMDA Research Grant Returns to Its Original Name in Honor of Tiffany L. House
The Acid Maltase Deficiency Association (AMDA) is renaming its research grant program back to the AMDA Research Grant, the name it carried when the program was first established in 2010. The change honors the life and legacy of Tiffany L. House, who passed away on May 25, 2025, and who served as president of the AMDA and chair of the International Pompe Association.
Application Documents & Instructions
Submit the completed LOI Cover Sheet and Letter of Intent as a single PDF by email to marzim50@gmail.com, named 2026-2027_LOI_Lastname.pdf.
Key Dates
Letters of Intent Due
October 15, 2026
Selected applicants will be invited directly to submit a full proposal following LOI review.
History & Legacy
2010
The program is established by the AMDA as the AMDA Research Grant, funding its first Pompe disease research project.
2013
Renamed the Helen Walker Research Grant, in memory of Helen Walker (1940–2012), founding president of the Australian Pompe Association and a board member of the International Pompe Association.
2026
The program returns to its original name, the AMDA Research Grant, honoring Tiffany L. House (d. May 25, 2025), former AMDA president and chair of the International Pompe Association.
Helen Walker’s legacy remains an essential part of the AMDA’s history, and her contributions to the global Pompe community continue to be honored in the AMDA’s work. Read Helen Walker’s full story →
AMDA Research Grant Recipients
2024 | Jaymin Upadhyay — Boston Children's Hospital
Jaymin Upadhyay, PhD
Boston Children’s Hospital, Harvard Medical School
Focus: Clinical outcomes and digital health monitoring
Title: Implementation of At-Home Functional Biomarker Methods in Patients with Pompe Disease
Summary:
This project seeks to develop simple, at-home tools that track how Pompe disease affects movement, breathing, speech, and thinking over time. By combining wearable sensors and biomarker tests, researchers aim to give patients and doctors an easier, more complete way to monitor health and treatment progress between clinic visits.
2023 | Gregory Wyant — Massachusetts General Hospital
Gregory Wyant, PhD
Massachusetts General Hospital, Harvard Medical School
Focus: Disease mechanism and therapeutic target discovery
Title: Autophagy Control of Glycogen in Skeletal Muscle
Summary:
This study is investigating how muscle cells normally break down glycogen inside lysosomes—the process that goes wrong in Pompe disease. By identifying the specific receptor that controls this step, researchers hope to uncover new targets for future therapies that could help restore healthy muscle function.
Learn More about the 2023 Award
2022 | Mark Tarnopolsky — McMaster University
Mark Tarnopolsky, BPE, MD, PhD
McMaster University Children’s Hospital
Focus: ERT optimization, biomarkers, and digital health innovation
Title: Biomarker and outcome measure discovery for Pompe disease through an evaluation of a Pompe Disease Targeted Multi-Ingredient Supplement (PDT-MIS)
Summary:
This project is working to make Pompe disease treatment more effective and personalized by identifying biomarkers that reflect disease activity and response to therapy. Researchers will also test wearable technologies and AI tools to improve how treatment progress is measured, while evaluating a new multi-ingredient supplement designed specifically for people with Pompe disease.
Learn More About the 2022 Award
2021 | Peter Meinke & Benedikt Schoser — LMU Munich
Peter Meinke, PhD, & Prof. Benedikt Schoser, MD
Friedrich-Baur-Institute, LMU Munich
Focus: ERT optimization and preclinical testing platform
Title: Establishing an in vitro platform for comparative analyses of therapeutic interventions and basic research in Pompe disease
Summary:
This project is creating a laboratory model to compare how different enzyme replacement therapies perform in muscle cells affected by Pompe disease. By testing multiple approved and experimental treatments side by side, researchers aim to better understand their effectiveness and develop faster, more accurate ways—like Raman spectroscopy—to measure treatment success at the cellular level.
Learn More About the 2021 Award
2019 | N.A.M.E. van der Beek et al. — Erasmus MC
N.A.M.E. van der Beek, MD, PhD, Prof. A.T. van der Ploeg, MD, PhD, & Prof. W.W.M. Pijnappel, PhD
Erasmus MC, Center for Lysosomal and Metabolic Diseases
Focus: ERT optimization and clinical outcomes
Title: Towards a [more] personalized treatment of patients with Pompe disease
Summary:
This international study aims to understand how patients respond differently to enzyme replacement therapy over the long term. By examining why some individuals benefit more than others, and whether higher doses or improved delivery could help, researchers hope to refine global treatment guidelines and make Pompe care more personalized and effective.
2018 | Emanuele Buratti & Andrea Dardis — ICGEB Trieste
Emanuele Buratti, PhD, & Andrea Dardis, PhD
ICGEB Trieste and Centro Malattie Rare, Udine, Italy
Focus: Therapeutic development (mutation-specific treatment)
Title: Preclinical validation of FDA approved molecules able to rescue GAA pre-mRNA splicing of c.-31-13T>G mutants as therapeutic agents for late-onset Pompe Disease
Summary:
This project is testing existing FDA-approved drugs that may correct the effects of a common genetic mutation found in many people with late-onset Pompe disease. By identifying small molecules that help cells process the enzyme properly, researchers hope to develop a targeted treatment that could improve or restore enzyme activity in affected patients.
Learn More About the 2018 Award
2017 | Virginia Kimonis — UC Irvine
Virginia Kimonis, MD
University of California, Irvine
Focus: Therapeutic development and ERT optimization
Title: Antisense oligonucleotide treatment for Pompe disease
Summary:
This research is testing a new treatment that targets the enzyme responsible for making glycogen in muscle cells. By using a genetic tool called antisense oligonucleotides (ASOs), scientists hope to reduce excess glycogen buildup—the root problem in Pompe disease—and strengthen muscles. The project will also explore how this new therapy works alongside current enzyme replacement therapy, aiming to create a more effective long-term treatment for patients.
Learn More About the 2017 Award
2016 | Frédéric LoFaso — Raymond Poincaré Hospital
Frédéric LoFaso, MD, PhD
Raymond Poincaré University Hospital, Garches, France
Focus: Clinical care and diagnostics (non-invasive monitoring)
Title: Acid Maltase Deficiency Myopathy Follow-Up
Summary:
This project aims to develop better, non-invasive ways to measure how Pompe disease progresses over time. By creating reliable tests that don’t require muscle biopsies or effort-based evaluations, doctors will be able to track patients’ health more easily and accurately—especially as new therapies become available.
Learn More About the 2016 Award
2015 | Nina Raben & Rosa Puertollano — NIH
Nina Raben, MD, PhD, & Rosa Puertollano, PhD
National Institutes of Health
Focus: Therapeutic development and ERT optimization
Title: Curcumin: Can it promote cellular clearance in Pompe disease?
Summary:
This study is exploring whether curcumin—a natural compound found in turmeric—can be safely added to current Pompe disease treatments. Researchers hope it may enhance the effects of enzyme replacement therapy or help protect muscle cells, offering patients improved treatment results.
2014 | Giancarlo Parenti — Federico II University
Giancarlo Parenti, MD
Federico II University, Naples, Italy
Focus: Diagnostics and biomarker discovery
Title: Analysis of Circulating and Tissue Specific MicroRNAs in Pompe Disease
Summary:
This project is using advanced genetic sequencing to identify small molecules called microRNAs that may serve as biomarkers for Pompe disease. These biomarkers could help doctors better diagnose the disease, track how well enzyme therapy is working, and even uncover new treatment targets to improve patient outcomes.
2013 | Barbara K. Smith — University of Florida
Barbara K. Smith, PhD, PT
University of Florida
Focus: Respiratory care and quality of life
Title: Response to Diaphragmatic Pacing in Subjects with Pompe Disease
Summary:
This study is evaluating whether diaphragm pacing—a device that helps stimulate the breathing muscles—can reduce the need for mechanical ventilation in adults with Pompe disease. Researchers will also assess whether this approach improves comfort, independence, and overall quality of life for patients who rely on breathing support.
2012 | Nuno Raimundo — University of Göttingen
Nuno Raimundo, PhD
University of Göttingen, Faculty of Medicine
Focus: Therapeutic development and disease mechanismTitle: Mitocho ndria and Autophagy as Therapeutic Targets for Acid Maltase Deficiency
Summary:
This project is testing new ways to boost the cell’s natural energy and cleanup systems—specifically mitochondria and autophagy—to lessen symptoms of Pompe disease. By strengthening these processes, researchers hope to improve how muscle cells function and uncover new treatment strategies beyond enzyme replacement therapy.
2011 | Pascal LaForêt — Pitié-Salpêtrière Hospital | Barbara K. Smith — University of Florida
Pascal LaForêt, MD
Pitié-Salpêtrière Hospital, Paris, France
Focus: Rehabilitation and quality of life
Title: Aerobic Exercise Training in Pompe Disease
Summary:
This study is testing whether regular aerobic exercise can help people with late-onset Pompe disease improve endurance, muscle strength, and breathing capacity. Understanding these benefits could help patients safely incorporate exercise into their care plan and enhance overall quality of life.
Learn More About this 2011 Award
Barbara K. Smith, PhD, PT
University of Florida
Focus: Respiratory care and neuromuscular function
Title: Clinical and Electrophysiological Assessment of Respiratory Function in Pompe Disease.
Summary:
This project aims to understand how nerve signals to the diaphragm—the main breathing muscle—are affected in adults with Pompe disease who have trouble breathing. By studying how nerve and muscle function relate to breathing ability, researchers hope to improve respiratory monitoring and guide better treatments to preserve breathing strength.
2010 | Andrea Amalfitano — Michigan State University
Andrea Amalfitano, DO, PhD
Michigan State University
Focus: Gene therapy development
Title: Pre-clinical studies for Acid Maltase Deficinecy (AMD) Gene Therapy in a Non-human Primate Model
Summary:
This project is exploring a new way to treat Pompe disease by delivering a healthy copy of the GAA gene directly to the liver using a gene therapy vector. If successful, the liver would produce and release the missing enzyme throughout the body, offering longer-lasting benefits than current enzyme infusions and potentially reducing the burden of frequent treatments for patients.
Acid Maltase Deficiency Association
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