Why Raising Funds for APBD Research Matters Now More Than Ever

We are excited to launch the 2025 APBD Tour de Friends Rally for Research fundraising campaign, aiming to raise $150,000.

Until May 16, your donations will be matched dollar-for-dollar, up to $50,000, by our generous Board.

Join Team APBD Tour de Friends Rally for Research in our effort to fund important APBD research, replacing UPenn’s Million Dollar Bike Ride (MDBR) campaign that we’ve participated in since 2016. We’ll be raising money until June 30 to support life-saving research.

Since our start in 2005, we’ve raised over $1.8 million for research grants through our Foundation and UPenn's MDBR. These grants have led to significant advancements in drug discovery and biomarker development initiatives that are progressing today. Despite the tough economic times, we’re dedicated to ensuring that APBD research advances and brings new therapies to patients.

Dr. Larry Arky Joins the APBD Research Foundation’s Board of Directors

The APBD Research Foundation is thrilled to welcome Dr. Larry Arky to its Board of Directors.



"Dr. Arky brings a unique perspective as both a physician and a patient living with this ultra-rare disease. He will help us elevate voices of the APBD patient community in a way that only someone with his background can. His involvement is invaluable as we continue our efforts to increase engagement, expand our reach, and make treatments a reality for those affected by APBD and allied diseases,” said Jeff Levenson, DDS, co-president.

Paving the Path to Treatments: Highlights from the APBD Biomarkers Workshop

On March 20, 2025, we hosted an APBD Biomarker Workshop bringing together over 20 researchers and doctors to tackle the urgent challenge of finding reliable biomarkers, which are measurable signs of disease.

The Workshop highlighted promising biomarker candidates (including GFAP, NfL, Tau, and short glucan chains) and explored the idea of a composite biomarker panel to track disease progression. Tracking how a disease evolves helps build a timeline of symptoms, progression rates, and key milestones, which are essential for developing targeted interventions and evaluating treatment effectiveness.

Accelerating Research with COMBINEDBrain Partnership



We’re excited to announce that the APBD Research Foundation is now a

member of the Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders (COMBINEDBrain).

By joining this group, we’re collaborating with over 92 organizations to accelerate the development of clinical treatments for people with rare genetic neurodevelopmental and neurodegenerative disorders by pooling efforts, studies, and data.

Key Takeaways from our Collaboration with Costello Medical

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We recently completed a collaboration with Costello Medical, a leader in medical communications and research, through its Pro Bono Call for Rare Disease Organizations. They helped us create important tools to connect the APBD and the broader GSD IV communities.

Together, we hosted two workshops, developed a strategic communications plan, and created an action roadmap to connect early- and late-onset GSD IV communities, increase awareness, improve education for providers, and enhance research engagement. This partnership is a significant step toward advancing research and therapy development in a more unified GSD IV community.

Editor’s Note: APBD (the late-onset form of GSD IV) and Andersen Disease (the early-onset form of GSD IV) share the same underlying root cause — changes in the GBE1 gene which causes deficiency of a protein called glycogen branching enzyme (GBE) in our body. These rare conditions are essentially different clinical manifestations of the same biochemical issue, making collaboration between our communities vital for progress.

Upcoming Events

Patient Chat

May 28, 2025 | 7pm ET | 4pm PT

The Patient Chat is hosted by volunteer moderator Harriet Saxe. To learn more, email Harriet at harriet@apbdrf.org.



Caregiver / Family Chat

May 8, 2025 | 7:30pm ET | 4:30pm PT

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

International Patient Chat

We're teaming up with UK-based organizations Alex TLC and AGSD-UK for a free virtual International APBD Patient Chat on June 25.

Register Now



This Chat offering aims to connect APBD patients globally, creating a community of support and research opportunities.

Thanks to Neuberger Berman for sponsoring this event.

Make a Legacy Gift to Fight APBD

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Have you thought about the lasting impact you can make on the APBD Research Foundation? Beth and Jeff Katz's journey with APBD inspired them to include the Foundation in their estate plan.

Beth shared, “As a family, we decided to leave a legacy of hope by including the APBD Research Foundation in our plan. This not only gives us peace of mind but also invests in a brighter future for everyone affected by this condition. Our experience with APBD has shown us the strength of community, and we’re proud to support the search for a cure.”

Make a lasting impact - now and beyond your lifetime…

Contact Anesa Kajtazovic at anesa@apbdrf.org with any questions.



P.S.: If you have already included a gift to the APBD Research Foundation in your estate plan, please fill out this form! We’d love to thank you for your generosity.



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 review your will and any other important legal documents FreeWill may generate for you.

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