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
Re-Scheduled–Friday, Oct 8, 2021: AMDA and Spark Webinar: Understanding Gene Therapy Research and the Potential Applications for Late Onset Pompe Disease
Hi All, We are excited to announce that the webinar is on for Friday, October, 8, 2021 at 1 PM CST. If you have already registered, you don't need to do anything. If you have NOT registered yet, it's not too late! Please click here to register. And, don't forget, we...
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Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease
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10th Annual PCMA Pull for Pompe!
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Audentes has released a Pompe Program Update. It includes information regarding FORTIS, their clinical trial for gene therapy for Pompe. To read the Pompe Program Update, please click here.
Spark Therapeutics Clinical Trial FAQ
Spark Therapeutics has developed a “Frequently Asked Questions” document regarding their RESOLUTE clinical trial. Please click here to read.
Patient Focused Drug Development Meeting—UPDATE
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2020 PCMA Pull For Pompe!—Cancelled
Update: July 8, 2020 It is with great sadness and disappointment that we hereby announce the cancellation of the 2020 Pull For Pompe Shoot due to the on-going COVID-19 pandemic. When we initially postponed the Shoot to August 15, we had high hopes that the virus would...
AMDA Q and A with Dr. Priya Kishnani on COVID-19 and Pompe
With all of the questions and uncertainty surrounding the ongoing COVID-19 pandemic, the AMDA worked with Dr. Priya Kishnani and her group at Duke University on the following Question and Answer Fact Sheet. We hope that the Pompe Community will find the information...
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
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San Antonio, Texas 78270 USA