TOP NEWS
Reports of Nexviazyme Shipment Delays
Pompe Alliance, Marshall’s Mountain, Grant’s Giants, Pompe Warrior Foundation, Ryan Colburn of the Pompe Community, and the AMDA are working together to help patients obtain clear and timely information regarding reports of NexviazymeA newer enzyme replacement therapy designed to improve delivery of the enzyme to muscle cells. shipment and release delays.
At this time, the delays being reported to our organizations involve some shipments of Nexviazyme. We have not received confirmation that LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme. is currently affected, but patients using either treatment may wish to verify their upcoming shipment.
This appears to be a product-release and distribution timing issue — not evidence of a safety or quality problem with medication that has already been released. Patients should not panic or make treatment changes without speaking with their prescribing medical team.
In June 2026, the FDA issued a warning letter following a January inspection of a Sanofi facility in Waterford, Ireland. Sanofi has publicly stated that products released from the facility meet established quality and safety specifications. The FDA warning letter does not specifically identify Nexviazyme or Lumizyme as affected products, and we cannot independently confirm the precise cause of every current shipment delay.
OTHER NEWS
2025 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 14th Annual PCMA's Pull for Pompe will take place on Saturday, April 26, 2025 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
2025 New Year Letter From AMDA President – Tiffany House
As we enter a new year, the Acid Maltase Deficiency Association (AMDA) wants to thank you for your unwavering support and dedication. Together, we have navigated through challenges and made significant strides for the Pompe community. Your support fuels our commitment...
2024 PCMA’s Pull for Pompe Photo Gallery
On Saturday, April 27, 2024, the PCMA (Precast Concrete Manufacturers Association) held it’s annual Pull for Pompe fundraiser at the National Shooting Complex in San Antonio, TX. The Pull for Pompe clay shooting event was a day of family fun designed for all ages and...
2024 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in 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. research. This grant opportunity was made possible by the 2024 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Essential Update: New Treatment Approaches for Late-Onset Pompe Disease
The AMDA is partnering with Medlive to bring this exciting program to HCPs in the Pompe community. Join leading experts Dr. Tahseen Mozaffar and Dr. Barbara Burton as they discuss late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly.. Hear the latest advancements, including newly approved...
2023 Helen Walker Grant Recipient
Project title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. control of glycogenA stored form of sugar used for energy. in skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. This year, the AMDA was fortunate to receive 13 grant applications focusing on both clinical research and basic science. It has been a challenge to select one. Dr. Wyant was chosen because he has embarked...
Join us for the first “Talking With Your Pompe Peeps” session on August 16, 2024!
We are excited to announce that the topic of our first "Talking With Your Pompe Peeps" session is "The Elementary School Experience: Building Success for IOPD/LOPD Children Through Collaboration and Partnership"! Join us August 16, 2024 at 1 PM CT for an open forum...
PEARL Trial-PrEnAtal enzyme Replacement for Lysosomal storage diseases
University of California - San Francisco (UCSF) is excited to announce the rebranding of their phase 1 clinical trail of In Utero Enzyme Replacement Therapy as the PEARL Trial-PrEnAtan enzyme Rreplacement for Llysosomal storage diseases. "We have rebranded our...
2024 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 13th Annual PCMA’s Pull for Pompe will take place on Saturday, April 27, 2024 at the National Shooting Complex in San Antonio, Texas! The Pull for Pompe clay shooting event is designed for all ages and ability levels. Please...
2023 Helen Walker Research Grant For Pompe Disease Now Accepting Applications
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by the 2023 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
Acid Maltase Deficiency Association
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