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

In Memory of Henri Termeer

he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known and worked with Henri since the late-90s through his role as the CEO of Genzyme. We will never forget how he always took the time to talk to patients and their...

read more

2017 PCMA of Texas Pull for Pompe!

The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday, March 25th at the National Shooting Complex in San Antonio, Texas! This is the 7th Annual Pull for Pompe hosted by the PCMA of Texas. The Pull for Pompe clay...

read more

2016 Helen Walker Research Grant Awarded

The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the Hôpital Raymond Poincaré in France for his Project: “Acid Maltase Deficiency Myopathy Follow-Up.” The aim of this project is to improve the...

read more

 Acid Maltase Deficiency Association

GET INVOLVED

Your generous contribution will go a long way into improving the quality of life of Pompe Disease Patients worldwide. Donations go towards Pompe Disease Research.

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