The APBD Research Foundation, in partnership with the Orphan Disease Center (ODC) at the University of Pennsylvania, is pleased to announce the launch of the 2024 Million Dollar Bike Ride (MDBR) Pilot Grant Program.
We are honored to be a part of this prestigious initiative, which offers up to 42 unique research grant opportunities across 34 rare diseases, including APBD. These funds were generously donated by our dedicated community during the Million Dollar Bike Ride on June 8, 2024.
Grant Details
The 2024 MDBR Pilot Grant Program provides a one‐year grant designed to propel APBD research forward.
The APBD Research Foundation is seeking research proposals such as:
- advancing the understanding of mechanisms of the disease, or
- clinical phenotyping that will facilitate future treatment trials, or
- basic science or clinical studies aimed at biomarker development (e.g., neurofilament light chain and glial fibrillary acidic protein assays) for the design of future therapeutic trials, or
- development of novel treatments.
Studies that have a strong likelihood of future federal funding are a plus.
Two grants are available at $56,857 each.
A project may be considered for up to $113,714 in funding if the researcher has an outstanding project and submits two proposals: one for $56,857 and another for $113,714.
The primary focus for this grant opportunity is the identification of a biomarker(s) that could be used to demonstrate the effectiveness of a therapeutic for APBD. Investigations related to the development of approaches that will prevent polyglucosan body accumulation or will facilitate its removal from the central and peripheral nervous systems will also be considered.
Applicants are encouraged to collaborate with other scientists and clinicians and should include a statement on resource sharing in their proposal. Applicants are encouraged to use existing disease models (i.e., mouse models, cultured skin fibroblasts) and to contact the APBD Research Foundation (info@apbdrf.org) with any questions about these resources. All grant applications will be considered confidential.
Application Process
We invite all eligible researchers to submit a Letter of Intent (LOI) by 8 PM EST on September 20, 2024. For detailed instructions on how to submit your LOI and to learn more about the application process, please visit the ODC website.
This Request for Applications (RFA) is open to the international research community. All individuals holding a faculty‐level appointment at an academic institution or a senior scientific position at a non-profit institution or foundation are encouraged to apply.
Please feel free to share this funding opportunity with your peers!
Check out our recently funded MDBR grant recipients and their research here:
- Or Kakhlon, PhD | Hadassah Medical Organization
Combinations of Small Molecule as Potential Therapeutic Agents for APBD - Priya Kishnani, MD, MBBS and Rebecca Koch, PhD, RDN, LDN | Duke University Medical Center
Deep Tissue and Cellular Phenotyping of APBD: A Bench to Bedside Approach - Matthew Gentry, PhD | University of Florida
Defining APBD Pre-Clinical Biomarkers and Assessing a Therapy in an APBD Mouse Model - Berge Minassian, MD and Mayank Verma, MD, PhD | UT Southwestern Medical Center
Glucan Fingerprint as a Biomarker in APBD - Wyatt Yue, PhD | Newcastle University, UK
Discovery of Glycogen Synthase Inhibitors for Validation as a Novel Therapeutic Target for APBD - Felix Nitschke, PhD | UT Southwestern Medical Center
Conquer from Within – Treating APBD by Viral Delivery of Cross-Correction-Enabled Amylase - H. Orhan Akman | Columbia University
Treatment of APBD Caused by an Intronic Mutation in the GBE1 Gene