Foundation Receives Patient and Family Support Grant from the Society for the Study of Inborn Errors of Metabolism
October 20th, 2020
The APBD Research Foundation has been awarded €4,500 ($5,270) by Europe-based Society for the Study of Inborn Errors of Metabolism. The award will be used to support patients and families impacted by APBD.
The SSIEM is a nonprofit organization committed to promoting interdisciplinary collaboration to advance the knowledge of inherited metabolic diseases.
“We are so grateful for the generous support of the SSIEM,” shares Harriet Saxe, Foundation Board member. Saxe adds, “This award increases our capacity to address our community’s great need for education, support, and wellness.”
The program—to be offered virtually in Fall/Winter 2020—will address a range of pressing topics: bladder dysfunction and the resulting impact; adapting to canes, rollators, scooters, and electric wheelchairs; understanding low energy and finding ways to cope; and caring for the APBD caregivers. Presentations will be made by subject matter experts.
By offering this innovative program in English, French, German, and Hebrew, the information that’s developed will reach many more in our global community. To get updates about this program and hear from the Foundation, subscribe to our newsletter.
News Releases
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We are thrilled to announce that our community raised an incredible $201,177 during the 2026 APBD Tour de Friends Rally for Research fundraising campaign, exceeding our original goal of $120,000.
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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.
The APBD Research Foundation is pleased to announce the third of our 2025 Rally for Research Pilot Grant Program grant recipients: Rebecca Koch, PhD, RDN at Duke University. Her research project is entitled, Gene Therapy to Treat the Neurophenotype of Glycogen Storage Disease Type IV.
The APBD Research Foundation is pleased to announce the second of our 2025 Rally for Research Pilot Grant Program grant recipients: Mayank Verma, MD, PhD at UTSW Medical Center. His research project is entitled Preclinical Validation and Biomarkers in APBD of a GYS1-ASO Currently in Phase 1 Clinical Trial for Lafora Disease
On behalf of the APBD community, the APBD Research Foundation has responded to the US Food and Drug Administration's (FDA) request for public comments for its recently released draft guidance for the use of the Plausible Mechanism Framework for individualized genetic therapies.
We are excited to launch the 2026 APBD Tour de Friends Rally for Research Fundraising Campaign to raise $150,000.
The APBD Research Foundation is excited to announce an opportunity for you to play a direct role in advancing treatment research. You do not have to leave home to participate.