Rachel’s Story

Rachel’s Story

May 18, 2026 | PATIENTS

May 2026 The following story was written by Courtney, Rachel’s daughter, and submitted to us to share her mother’s experience. We are grateful for her willingness to give us this window into their family’s journey. While we strive to share accurate...
Let’s Talk Diet — The Real Talk

Let’s Talk Diet — The Real Talk

May 1, 2026 | UPCOMING TWYPP SESSIONS

Webinar Details Title: Let’s Talk Diet — The Real Talk Date: Thursday, May 28, 2026 Time: 12 p.m. CT / 1 p.m. ET Moderator:The AMDA staff Webinar Overview:We all know eating well matters, but let’s be honest, actually sticking to a diet is hard. Life gets...
Move How You Can, Rest All You Need

Move How You Can, Rest All You Need

Apr 2, 2026 | ARCHIVES, ARTICLES

Move How You Can, Rest All You Need Hey, all! Lucas Garrett, here. As we head into April, I wanted to take a minute to jot down a few ideas on staying healthy — physically and mentally. As many in the Pompe community may already be keenly aware, well-regulated habits...
Immune Responses to AAV Gene Therapy

Immune Responses to AAV Gene Therapy

Mar 30, 2026 | PAST WEBINARS

Webinar Details Title: Immune Responses to AAV Gene Therapy: Challenges and Emerging Solutions Date: Thursday, April 23, 2026 Speaker:Abigail Benkert, MD Webinar Overview:Immune responses to AAV gene therapy remain a major barrier to its access and effectiveness. This...
Home Infusions

Home Infusions

Mar 25, 2026 | PAST TWYPP SESSIONS

Session Details Title: Home Infusions Date: Wednesday, April 29, 2026 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...
Tiffany House Receives RareVoice Award for State Advocacy

Tiffany House Receives RareVoice Award for State Advocacy

Feb 5, 2026 | ARCHIVES, NEWS

We are honored to share this moment recognizing Tiffany House as a recipient of the RareVoice Award for State Advocacy. The award is presented by the EveryLife Foundation For Rare Diseases. This award is given to advocates whose voices have helped shape meaningful...
2026 PCMA Pull for Pompe Fundraiser

2026 PCMA Pull for Pompe Fundraiser

Feb 4, 2026 | ARCHIVES, NEWS

The AMDA is excited to announce that the 15th Annual PCMA’s Pull for Pompe fundraiser will take place on Saturday, May 2, 2026, from 8 a.m. to 2 p.m. CST at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed...