APBD Research Foundation 2025 Rally for Research Grants Awarded to UCLA, UTSW Medical Center, and Duke University Scientists
May 19, 2026
The APBD Research Foundation awarded three 2025 Rally for Research Pilot Grants in 2026 to APBD researchers: Gal Bitan, PhD and Brent Fogel, MD, PhD at University of California, Los Angeles), Mayank Verma, MD, PhD at UTSW Medical Center, and Rebecca Koch, PhD, RDN at Duke University.
From left to right: Dr. Mayank Verma, Dr. Gal Bitan, Dr. Brent Fogel, and Dr. Rebeca Koch
Every year, the APBD Research Foundation works to raise funds to support pilot grants from scientific and clinical researchers. In 2025, the APBD Rally for Research campaign raised $150,000 from 287 donors to support 1-year APBD pilot research studies.
Drs. Gal Bitan and Brent Fogel | University of California, Los Angeles
Research Grant Project: New Biomarkers for Adult Polyglucosan Body Disease
Dr. Bitan and Fogel’s proposal seeks to identify biomarkers that can improve the diagnosis of APBD and capture disease progression, as well as be used as outcome measures to determine the effectiveness of new therapies. The study will use a highly sensitive protein assay to measure blood-based biomarkers that point to changes in the brain in patients with APBD, early-onset GSD IV, and APBD patients who have received the GHF-201 drug through Israel’s compassionate use program.
Dr. Mayank Verma | UTSW Medical Center
Research Project: Preclinical validation and biomarker in APBD of a GYS1-ASO currently in Phase 1 for Lafora Disease
With this research grant, Dr. Verma seeks to evaluate whether an antisense oligonucleotide (ASO) that reduces glycogen synthase (GYS1) developed for Lafora Disease, ION283, can reduce polyglucosan bodies and serve as a treatment for APBD. Glycogen synthase is the enzyme encoded by the GYS1 gene and is involved in the accumulation of polyglucosan bodies.
Dr. Rebecca Koch | Duke University
Research Grant Project: Gene Therapy to Treat the Neurophenotype of Glycogen Storage Disease Type IV
Dr. Koch’s study aims to evaluate the potential for nervous system-targeted delivery of gene replacement therapy to treat APBD and GSD IV. Gene replacement therapy is a type of gene therapy that delivers a functional copy of the affected gene - GBE1 for APBD and GSD IV - to help make more of the necessary protein - here, glycogen branching enzyme. This type of therapy is not specific to a certain genetic variant or mutation which means that it has the potential to treat any patient with APBD or GSD IV.