Our Mission
The Acid Maltase Deficiency Association’s driving force lies in its efforts to advance research, improve care, and ensure early diagnosis for individuals and families affected by Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. More. Founded by the House family and guided by individuals living with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. More, AMDA works through deep collaboration with global experts, industry, and policymakers to translate science into informed, compassionate support for the Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. More community.
What is Pompe disease?
Research
Webinars
News
Conferences
Recent News
Honoring Advocacy: Celebrating Tiffany House and the RareVoice Awards
This week is Rare Disease Week, with...
Tiffany House Receives RareVoice Award for State Advocacy
We are honored to share this moment...
2026 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that...
With Gratitude: Honoring Our 2025 Donors
The Acid Maltase Deficiency...
Tiffany House and Maryze Schoneveld van der Linde Honored at Sanofi’s Global Rare Impact Awards
At Sanofi’s inaugural Global Rare...
Grief and Rare Disease: Recognizing Loss and Building Grief Literacy
This past May, our hearts broke twice....
Recent Blog Posts
Move How You Can, Rest All You Need
Hey, all! Lucas Garrett, here. As we...
New Year’s Resolutions and Hobbies
For many, the end of the calendar year...
Burnout, Creativity, and Rest
For me, music can be one of the best...
Morgan’s Story: When Hope Met Hurricane Katrina
Editor's NoteIn the early years of...
Upcoming
Webinar
Immune Responses to AAV Gene Therapy
Title: Immune Responses to AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cells. Related Terms: Gene therapy; Vector (gene therapy vector); Redosing; Immune response More Gene Therapy: Challenges and Emerging Solutions
Date: Thursday, April 23, 2026
Time: 12 p.m. CST / 1 p.m. EST
Speaker:
Abigail Benkert, MD
Summary:
Immune responses to AAVA harmless virus commonly used as a delivery system in gene therapy to carry healthy genes into cells. Related Terms: Gene therapy; Vector (gene therapy vector); Redosing; Immune response More gene therapy remain a major barrier to its access and effectiveness. This talk will highlight key immune challenges and emerging strategies to overcome them, including antibody-cleaving enzymes and novel capsids designed to evade pre-existing immunity.
Upcoming Talking With Your Pompe Peeps Session
Home Infusions
Title: Home Infusions
Date: Wednesday, April 29, 2026
Time: 7 p.m. CST / 8 p.m. EST
Moderator:
Ashley Lloyd, BSN, RN
Summary:
Home infusions can feel overwhelming at first. Join Ashley Lloyd, BSN, RN, for a practical discussion on how to prepare, what to expect, and ways to make infusion days run more smoothly.
Get Involved
Your generous contribution will go a long way into improving the quality of life of Pompe Disease Patients worldwide. Donations go towards Pompe Disease Research.
Acid Maltase Deficiency Association
For the AMDA videos: All Rights reserved. No part of the AMDA's videos may be reproduced or transmitted in any form or by any means, electronic or mechanical, without the written permission of the copyright holder.
If you are interested in learning more about Pompe Disease and would like to make a contribution in support of necessary research, please contact us at:
info@amda-pompe.org
THE AMDA
PO Box 700248
San Antonio, Texas 78270 USA