JUST PUBLISHED! New Study Highlights That Over 34,000 People Worldwide Are Affected by APBD and Other Forms of GSD IV

Why Raising Funds for APBD Research Matters Now More Than Ever

The APBD Research Foundation is launching the 2026 APBD Tour de Friends Rally for Research fundraising campaign, aiming to raise $120,000.

Through May 15, your donations will be matched dollar-for-dollar,

up to $50,000, by our Board of Directors.

Join Team APBD Tour de Friends Rally for Research as we work to fund important APBD research, replacing UPenn’s Million Dollar Bike Ride (MDBR) campaign which we participated in from 2016 through 2024. We will be raising funds until June 30 to support life-saving research.

These funds are critical to advancing research studies and preparing our community for clinical trials -- key steps that bring us closer to effective treatments for APBD patients.

A FEW DAYS LEFT: Participate in the Drug Repurposing Program

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Have you been diagnosed with APBD or early-onset GSD IV (Andersen Disease)?

This is your chance to be part of a groundbreaking drug repurposing study with one goal: Find an existing drug that could help treat APBD. 



Deadline May 1st.

Take the next step today!

Foundation Submits Public Comment to the FDA on the Mechanism for Accelerating Individualized Therapy Development for Rare Diseases

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Earlier this year, the US Food and Drug Administration (FDA) released draft guidance to address the regulatory pathway(s) for developing individualized therapies for rare diseases. The FDA’s “Plausible Mechanism Framework” outlines considerations for the drug development process – particularly for gene and RNA-based therapies, such as gene editing* and antisense oligonucleotides* (ASOs) – used in rare diseases with small patient populations.

The APBD Research Foundation submitted comments to the FDA on this draft guidance to support the APBD and GSD IV communities and to help ensure that patient voices are included in shaping future therapies.

APBD Researchers Present on “GHF-201” at the AAN’s Annual Conference

From left to right: Dr. Or Kakhlon, Dr. Yossi Gilgun-Sherkim (GHF), Dr. Michio Hirano, and Yaniv Glazer (GHF)

The American Academy of Neurology's (AAN) Annual Conference in Chicago, IL featured presentations from Michio Hirano, MD (Columbia University) and Or Kakhlon, PhD (Hadassah Medical Center) highlighting their work in APBD. Both presentations centered around Golden Heart Flower’s investigational drug, GHF-201*, in development for APBD:

• Dr. Kakhlon’s talk focused on the outcomes of the Urgent Compassionate Use Program in Israel

• Dr. Hirano’s poster presentation focused on the proposed framework for a future Phase 2/3 clinical trial* of GHF-201 in APBD patients.

Upcoming Events

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

Patient Chat

May 27, 2026 | 9am PT | 12pm ET 

June 24, 2026 | 9am PT | 12pm ET

The hour will also include open mic time to discuss your discoveries, questions, and other thoughts.

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

Caregiver / Family Chat

May 14, 2026 | 9:30am PT | 12:30pm ET

June 11, 2026 | 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.

*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!