Foundation Launches Drug Repurposing Initiative in Partnership with COMBINEDBrain
June 17, 2026
In April 2026, the APBD Research Foundation announced participant recruitment for its APBDRF Drug Repurposing Study initiative in partnership with COMBINEDBrain. We are grateful to the patients and families in our community that have volunteered to provide biosamples to help make this study a reality.
Making treatments a reality for the Glycogen Storage Disease type IV (GSD IV) community, including early-onset GSD IV and adult-onset GSD IV (or Adult Polyglucosan Body Disease), is a critical goal of the APBD Research Foundation. The drug repurposing initiative is a parallel strategy to the traditional drug discovery efforts that the Foundation has invested over $2 million dollars since its founding in 2005.
Check out our inaugural podcast - Episode 1: The Treatment May Already Be Out There - to learn more about this initiative and how it may help bring treatments to the 34,000+ patients in the world that may be affected by GSD IV.
Using nasal swabs collected from 30 APBD and early-onset GSD IV patients and unaffected family members, the study aims to identify existing drugs that have been approved for other health conditions that have the potential to benefit our patient community.
How does drug repurposing help the GSD IV community? The benefit of drug repurposing is that approved drugs already have a wealth of information collected on their safety, dosing, what parts of the body it can treat, potential side effects, and how the drug works. This means that one of the steps of the drug approval/clinical trial process has already been completed -- compressing the timeline and costs significantly to get the drug to patients sooner.
For additional details on the study, see our recruitment page: We Need You for the APBDRF Drug Repurposing Study!
Interested in helping us underwrite the cost of expanding our APBDRF BioBank initiative? Please email Lindsay Gill, PhD at lindsay@apbdrf.org.