Rally for Research Campaign
In 2025, the APBD Research Foundation launched the Rally for Research campaign dedicated to advancing APBD research. The annual campaign builds on nearly a decade of participation in the Penn Medicine Orphan Disease Center’s Million Dollar Bike Ride (MDBR), which we partnered with from 2016 through 2024.
Since our founding in 2005, we have raised and awarded over $2 million in research grants, both directly as a Foundation and through our partnership with UPenn’s MDBR. Recipients include researchers at Columbia University, Duke University Medical Center, UCLA, UT Southwestern Medical Center, the University of Florida, Newcastle University, and Hadassah Medical Organization.
These seed grants have helped lay the foundation for the groundbreaking drug discovery and biomarker development initiatives progressing today.
Check out the funded grant recipients and their research here:
2025
New Biomarkers for Adult Polyglucosan Body Disease
Gal Bitan, PhD & Brent Fogel, MD, PhD (UCLA)Preclinical Validation and Biomarkers in APBD of a GYS1-ASO Currently in Phase 1 Clinical Trial for Lafora Disease
Mayank Verma, MD, PhD (UTSW Medical Center)Gene Therapy to Treat the Neurophenotype of Glycogen Storage Disease Type IV
Rebecca Koch, PhD, RDN (Duke University)
2024
From Mouse to Human – Establishing Novel Biomarkers for APBD
Felix Nitschke, PhD (UTSW Medical Center)Development of Glycogen Synthase Inhibitors for APBD
Wyatt Yue, PhD (Newcastle University)
2023
Combinations of Small Molecule as Potential Therapeutic Agents for APBD
Or Kakhlon, PhD (Hadassah Medical Organization)Deep Tissue and Cellular Phenotyping of APBD: A Bench to Bedside Approach
Priya Kishnani, MD, MBBS and Rebecca Koch, PhD, RDN, LDN (Duke University Medical Center)
2022
Defining APBD pre-clinical biomarkers and assessing a therapy in an APBD mouse model
Matthew Gentry, PhD (University of Florida)Glucan fingerprint as biomarker in APBD
Berge Minassian, MD and Mayank Verma MD, PhD (UT Southwestern Medical Center)
2021
Discovery of Glycogen Synthase Inhibitors for Validation as a Novel Therapeutic Target for APBD
Wyatt Yue, PhD (Newcastle University)
2020
Conquer from Within: Treating APBD by Viral Delivery of Cross- correction-enabled Amylase
Felix Nitschke, PhD (UT Southwestern Medical Center)
2019
Testing the Efficacy of New Pharmaceutical Candidates for APBD
Or Kakhlon, PhD (Hadassah Medical Organization)Gene Therapy for APBD
Baodong Sun, PhD (Duke University)
2018
Pharmacological, Histological and Metabolic Analyses of a New Drug Candidate for APBD
Or Kakhlon, PhD (Hadassah Medical Organization)Gene Replacement Therapy for APBD
Berge Minassian, MD (UT Southwestern Medical Center)
2017
Could Restoring GBE in Affected Tissues be a Treatment for APBD?
H. Orhan Akman, PhD (Columbia University Medical Center)Testing APBD Drug Candidates for Restoration of Multiple Cell Features and Gene Expression Profile
Or Kakhlon, PhD (Hadassah Medical Organization)
2016
The Efficacy of Guaifenesin in the Treatment of APBD
H. Orhan Akman, PhD (Columbia University Medical Center)Treatment of APBD Caused by an Intronic Mutation in Glycogen Branching Enzyme Gene
H. Orhan Akman, PhD (Columbia University Medical Center)