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 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 12th Annual PCMA’s Pull for Pompe will take place on Saturday, April 29, 2023 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...
2022 Helen Walker Research Grant For Pompe Disease Now Accepting Appliations
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 2022 Pull for Pompe, a private fundraiser held by the Precast Manufacturer's Association of Texas...
An Open Letter to the Pompe Community
On October 28, Amicus Therapeutics issued a press release with an update on the progress of our Pompe program as we received information from the US Food & Drug Administration (FDA) on the regulatory status of our next-generation therapy for the treatment of...
International Survey on Pompe Disease and Covid-19
Over the last 2 years the COVID-19 pandemic has had wide reaching effects on people all over the world. To better understand the impact of the Covid 19 Pandemic on the worldwide Pompe community, the International Pompe Association and the Erasmus MC are undertaking a...
2021 Helen Walker Grant for Pompe Disease Winner Announcement
The AMDA is excited to announce that the 2021 Helen Walker Grant for Pompe Disease was awarded to Dr. Peter Meinke and Dr. Benedikt Schoser for their Project entitled: "Generation of a platform for comparative testing of new treatments for people living with Pompe...
Astellas Gene Therapies Press Release – Clinical Trial Update
"On June 26, 2022, Astellas Pharma, Inc. ("Astellas") issued a press release announcing that the U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 clinical trial evaluating AT845 ("the investigational gene therapy candidate")...
Voice of the Patient Report for the Pompe PFDD is Released!
The AMDA is excited to report that the final Voice of the Patient Report for the Pompe Patient-Focused Drug Development meeting has been released. To view the report, please CLICK HERE!
Aro Biotherapeutics to Present New Preclinical Data
Aro Biotherapeutics to Present New Preclinical Data Highlighting the Potential of Centyrin-siRNA Conjugates for the Treatment of Pompe Disease. To read the article, please click here.
2022 PCMA Pull for Pompe Fundraiser
The AMDA is excited to announce that the 11th Annual PCMA’s Pull for Pompe will take place on Saturday, April 30, 2022 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...
2021 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 2021 Pull for Pompe, a private fundraiser held by the Precast Manufacturer’s Association of Texas...
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