Acid Maltase Deficiency Association - AMDA

04/21/2026

Gene therapy continues to be one of the most promising areas of research in Pompe disease, but immune responses to AAV vectors remain a major challenge. This webinar will break down what researchers are learning and the innovative strategies being developed to overcome these barriers, including approaches like antibody-cleaving enzymes and next-generation vectors.

This is an important opportunity to better understand where the science stands today and what it could mean for future treatment options.

New Date: Thursday April 23
Time: 12 PM CT

Make sure you are registered so you don’t miss it.https://us06web.zoom.us/webinar/register/WN_qwO2v77xQ1C36nuBOch2Aw #/registration

Pompe disease is rare, support shouldn’t be.

04/17/2026

Most people go into the weekend thinking about everything they want to do.

But with Pompe disease, it often starts with something else entirely; how much energy do I actually have to work with?

Instead of trying to fit everything in, many in the Pompe community think in terms of an energy budget.Energy is not unlimited and once it is spent, it is not easily regained.

That might look like:
• Choosing one or two meaningful activities instead of a packed schedule
• Planning rest before and after outings
• Leaving margin instead of running on empty
• Saying no to something good so you can say yes to something better

It is not about doing less, it is about spending your energy with intention.
As the weekend approaches, ask yourself: What is actually worth my energy?

We would love to hear from you.
What is one thing you are choosing to spend your energy on this weekend?

Pompe disease is rare, support shouldn’t be.

04/16/2026

Accessing care for Pompe disease is rarely straightforward.
From the first signs of symptoms to diagnosis and long-term management, the process can feel overwhelming and, at times, uncertain.

Many individuals and families face:
• Delayed or complex diagnoses due to overlapping symptoms
• The need to coordinate multiple specialists and therapies
• Questions around insurance, referrals, and ongoing care
• The challenge of advocating for the right support network

Because Pompe disease is rare, not every provider has experience treating it. That makes preparation, education, and coordination essential.

A few key steps can make a meaningful difference:
• Preparing for appointments with symptom history and questions
• Understanding diagnostic testing options like enzyme and genetic testing
• Building a multidisciplinary care team that communicates effectively
• Tracking symptoms, treatments, and progress over time
You don’t have to navigate this alone.

AMDA is here to help guide you through each stage, offering trusted information and support along the way.
Read the full resource herehttps://amda-pompe.org/wp-content/uploads/2024/04/Accessing-Care-and-Resources-Final-post-04.05.24.pdf

Because Pompe disease is rare. Support shouldn’t be.
What part of the care journey has been the most challenging for you or your family?

04/15/2026

nternational Pompe Day is more than a date; it is a global movement uniting voices, strength, and hope across the world. On this day, we are reminded that every move counts; not just today, but every single day.

As we come together in the Walk, Run, and Roll event with the International Pompe Association (IPA), we honor the resilience of those living with Pompe, the dedication of caregivers, and the commitment of advocates advancing awareness and progress.

The AMDA proudly stands in this observance, not just on April 15, but every day. Because for us, it's more than a moment — it's a mission:

P - Passionate
O - On
M - Matters
P - Pompe
E - Everyday

Together, we move forward, with purpose, with passion, and with each other. 💜

With purpose, passion, and community,

The Acid Maltase Deficiency Association

04/14/2026

The Precast Concrete Manufacturers Association (PCMA) invites you to enjoy a family-friendly sporting clay event that welcomes participants of all ages and experience levels.

Whether you are an experienced shooter or simply looking to try something new, this event offers an opportunity to spend meaningful time with others while enjoying a unique outdoor activity.

This event benefits the Acid Maltase Deficiency Association (AMDA), which has been dedicated since 1995 to advancing research and increasing awareness for Pompe disease.

Although treatment is now available, there is still no cure. Support from events like this helps fund research that continues to improve outcomes for patients and move the field forward.
We are grateful for your support and hope you will join us. Register or donate at the link below.

https://pullforpompe.org/product/event-registration/

Pompe disease is rare, support shouldn’t be.

04/10/2026

This webinar has been postponed. Check back for new time

This is happening Friday at 12 PM CT and you’ll want to be in the room for it.

Gene therapy is advancing quickly in Pompe disease, but one major challenge remains: the body’s immune response to AAV therapy.

Researchers are now developing real solutions to overcome this barrier, and it could change what’s possible for future treatment.
Join us for:
Immune Responses to AAV Gene Therapy: Challenges and Emerging Solutions

Led by Abigail Benkert, MD of Duke University Medical Center, this webinar will break down:
• Why immune responses matter
• What’s being done to solve it
• What it could mean moving forward

👉 Spots are filling. Register now: https://amda-pompe.org/immune-responses-to-aav-gene-therapy/

Pompe disease is rare. Support shouldn’t be.

04/10/2026

This is what’s actually helping people with Pompe right now-
Small routines people are using every day.

At-home infusions when possible
Telehealth check-ins
Pre-hydrating before infusion days
Keeping a “go bag” ready
Pacing energy instead of pushing to exhaustion
Planning rest before burnout hits
Short, consistent stretching or movement
Tracking symptoms or energy patterns

These aren’t flashy but they’re helping people take back time, energy, and control.

What’s one routine that’s made your day-to-day life easier? Drop it below

04/08/2026

This is happening Friday at 12 PM CT and you’ll want to be in the room for it.

Gene therapy is advancing quickly in Pompe disease, but one major challenge remains: the body’s immune response to AAV therapy.

Researchers are now developing real solutions to overcome this barrier, and it could change what’s possible for future treatment.
Join us for:
Immune Responses to AAV Gene Therapy: Challenges and Emerging Solutions

Led by Abigail Benkert, MD of Duke University Medical Center, this webinar will break down:
• Why immune responses matter
• What’s being done to solve it
• What it could mean moving forward

👉 Spots are filling. Register now: https://amda-pompe.org/immune-responses-to-aav-gene-therapy/

Pompe disease is rare. Support shouldn’t be.

04/07/2026

If you wait to feel motivated… you may never start

Let’s be honest, motivation doesn’t always show up.

Especially when you’re living with Pompe disease, there are days when movement feels hard, exhausting, or just not appealing at all.

But here’s the truth many people learn over time, you don’t wait for motivation.
You move first… and motivation often follows.

Movement doesn’t have to be big to matter.
Some days it’s stretching.
Some days it’s a short walk.
Some days it’s simply choosing to move your body in any way you can.
Those small decisions add up.

At AMDA, we see how consistency, not perfection, plays a key role in helping individuals maintain strength and function over time.
What helps you get moving on the days you don’t feel like it?
Pompe disease is rare, support shouldn’t be.

04/01/2026

Each year, International Pompe Day brings the global Pompe community together through one powerful idea: movement with purpose.

The Run Walk Roll initiative, led by the International Pompe Association, invites individuals and families to move in whatever way they can. Every step, stride, or wheel forward represents strength, resilience, and awareness for Pompe disease.

But this event goes beyond movement.

Participants have the option to raise funds, and those donations are directed toward a patient survey study at Erasmus Medical Center Rotterdam, The Netherlands. This research helps the medical community better understand the real, day-to-day experiences of people living with Pompe disease.

Why this matters:�• It captures patient-reported outcomes and lived experiences�• It helps inform future care and treatment approaches�• It strengthens global understanding of Pompe disease beyond clinical settings

If you’d prefer to raise funds to support the work we’re doing at AMDA, you are absolutely welcome to do that as well. Every effort helps strengthen this community and expand access to meaningful resources, education, and support.

If you’re participating this year, we’d love to hear how you’re choosing to Run, Walk, or Roll. Share your experience below and help inspire others to get involved.

Every move helps muscles move and creates awareness of this rare disorder.

03/31/2026

New Direction in Gene Therapy?

What if gene therapy could work around one of its biggest challenges… the body’s immune response?

Join us on April 10 at 12 PM CT for a deep dive into emerging research on AAV gene therapy, including a novel “duck-derived” viral vector that may help overcome pre-existing immunity, one of the biggest barriers in treatment today.

We’re honored to welcome Dr. Abigail Benkert, MD, who will walk us through:
• Why immune responses limit current AAV gene therapies
• What makes this new “duck vector” approach different
• What this could mean for future treatment strategies in Pompe disease

This is cutting-edge science, explained in a way that helps our community stay informed, ask better questions, and better understand what’s ahead.

📅 April 10 | 12 PM CT
🔗 Register now and stay connected to the latest in Pompe research https://amda-pompe.org/immune-responses-to-aav-gene-therapy/

03/30/2026