2025 Rally for Research Series: UTSW Medical Center and Duke University Awarded Pilot Grants

The APBD Research Foundation has awarded its second and third 2025 Rally for Research pilot grants to UTSW Medical Center researcher Dr. Mayank Verma and Duke University researcher Dr. Rebecca Koch.

Preclinical* Validation and Biomarkers* in APBD of a GYS1-ASO Currently in Phase 1 for Lafora Disease

With this grant, Dr. Verma aims to evaluate the effectiveness of ION283, an antisense oligonucleotide therapy that reduces glycogen synthase* levels, as a treatment for APBD. Read More

Gene Therapy* to Treat the Neurophenotype of Glycogen Storage Disease Type IV*

With this grant, Dr. Koch aims to evaluate the effectiveness of nervous system-targeted delivery of gene replacement therapy* to treat APBD and other forms of GSD IV. Read More

With these awards to Dr. Mayank Verma, and Dr. Rebecca Koch -- as well as to Dr. Gal Bitan announced in February -- the APBDRF has now awarded three 2025 Rally for Research grants totaling $150,000. Our thanks to each of you -- Board members, patients, family members, and friends -- for your commitment to advancing research!  

What is the #Findthe34000 Campaign?

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The Find The 34,000 campaign is about addressing the gap between the ~346 GSD IV -- including early-onset GSD IV and adult-onset GSD IV (APBD) -- patients diagnosed worldwide and the new prevalence study's 34,000+ global estimate for all individuals with GSD IV.

Be sure to check out our recently published flyer for healthcare professionals and patients, as well as other resources aimed at helping build strength, community, and power in numbers.

Check Out Our New Flyers

JUST IN -- Second Match Challenge to Boost Our 2026 Rally for Research Fundraiser

We are thrilled to announce that Dr. Ora Gordon has created a second matching opportunity for $20,000, building on our Board match earlier in the 2026 Rally for Research campaign. 

P.S.: If you have already made a gift, thank you!

Thank you for your commitment to making research advancements and treatments possible!

Upcoming Events

The Foundation hosts talks, workshops, and scientific meetings for patients, caregivers, and health professionals. These programs connect our community members and deepen the understanding of how APBD impacts lives.

• Team Knoll Bike Ride | Austin, TX

June 7 (8am CT / 9am ET)

Community member Allan Rosenthal is hosting his second annual bike ride in Austin (TX) in honor of Mark Knoll and the APBD community. If you’re in the Austin area or have friends and family there, help spread the word and join this exciting ride in support of APBD research!



To learn more, email Anesa Kajtazovic, our Community Affairs Manager, at anesa@apbdrf.org.

• Caregiver / Family Chat

June 11 (4:30pm PT / 7:30pm ET) 

This Chat is hosted by volunteer moderator Linda Cedarbaum. To learn more and register for this event, email Linda at linda@apbdrf.org.

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• Virtual Community Game Night

June 23 (5pm PT / 8pm ET)

Join the APBDRF team for a light-hearted evening of community fellowship and games on June 23 (8pm ET). Together, we will celebrate the success of the APBD Tour de Friends Rally for Research campaign and the community we are building. Spots are limited to the first 100 registered participants. Register here...

• Patient Chat

June 24 (9am PT / 12pm ET) 

This Chat is hosted by volunteer moderator Harriet Saxe. To learn more or register for this event, email Harriet at harriet@apbdrf.org.

May is Leave a Legacy Month. What Will Yours Be?

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The future of APBD research is being shaped by the people who believe in hope, progress, and making a lasting impact.

During the month of May, which is designated as “Leave a Legacy Month,” we invite you to create your will through FreeWill -- a free, easy-to-use resource -- that helps you protect your loved ones and support causes that matter deeply to you.



A legacy gift to the APBD Research Foundation can help advance research, support families, and move us closer to treatments for generations to come. In as little as 20 minutes you can create your free will.

For questions about FreeWill or legacy giving, contact Anesa Kajtazovic at anesa@apbdrf.org.



Disclaimer: Please note that FreeWill is not a law firm and cannot provide legal advice, nor can the APBD Research Foundation. We would always encourage that a lawyer reviews your will and any other important legal documents FreeWill may generate for you.

*Glossary

The glossary provides definitions for the words highlighted in grey in this newsletter.

We provide this FREE monthly newsletter resource to all of our GSD IV and allied disease community members. This resource is made possible by contributions to the APBD Research Foundation from donors like you. Thank You!