2023 Helen Walker Grant Recipient
August 1, 2024
Project title: Autophagy control of glycogen in skeletal muscle
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 on a discovery project aimed at solving a missing piece of the puzzle: how does glycogen enter the lysosomes? A presumptive pathway for glycogen traffic to the lysosome is autophagy – a familiar term in Pompe disease research. In fact, one of the canonical images describing the disease illustrates glycogen transport to the lysosome in a vesicle (autophagosome) that is formed during autophagy. Although the concept of glycogen autophagy, called glycophagy, recently gained a lot of interest, the mechanism of muscle glycogen entrapment into the autophagosome has eluded the researchers for a long time.
The team developed a novel sophisticated approach to directly follow glycogen trafficking to the lysosome in skeletal muscle. The proposed study holds promise to identify a protein(s) that bind to glycogen and anchors it to the autophagosomal membrane. The implications of this discovery could be manipulated, so that less glycogen is delivered to the lysosomes, thus limiting damage to the muscle cells.
The application scored highest for novelty and scientific depth. We welcome Dr. Wyant and his team to the Pompe community.
OTHER NEWS
2023 Helen Walker Grant Recipient
Project title: Autophagy control of glycogen in skeletal muscle 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...
Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease
PHILADELPHIA, Sept. 28, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is...
CALL to participate in IPA Project Every Move Counts
At International Pompe Day the International Pompe Association kicked off the Every Move Counts project. This project focuses on raising awareness of the importance of movement. We all have different abilities and levels of disability, but the important thing to...
2024 Acid Maltase Deficiency Association (AMDA)/International Pompe Association (IPA) Conference date has been set!
Dear Pompe Warriors and Patient Community, We are excited to announce the upcoming 2024 Acid Maltase Deficiency Associate - International Pompe Association (AMDA-IPA) Conference, a momentous event dedicated to advancing knowledge, support, and advocacy for individuals...
Rare Artist Contest is now Open for Submissions
Enter July 23rd - August 31st at RareArtist.org The Rare Artist Program was established in 2010 to exhibit the unique gifts of individuals impacted by rare disease to tell their story through art. Now in year 13, the Rare Artist Annual Contest is focused on providing...
Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the European Union
PHILADELPHIA, June 27, 2023 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced that the European Commission (EC) has...
Acid Maltase Deficiency Association
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
AMDA
PO Box 700248
San Antonio, Texas 78270 USA