Our APBD Tour de Friends Rally for Research fundraiser was a huge success! We surpassed our goal of $150,000 and raised $178,404.  Thank you to all 287 donors, our Board of Directors who matched $50,000, and Dr. Ora Gordon, who matched $36,000. We also thank our peer-to-peer fundraising team leaders: Kathy Arky; Melanie Robinson, Faye Rosenberg, Alan Rosenthal and Robin Knoll, Carol Rosenstock, and Sharon Young. 

In this newsletter, we highlight two powerful examples of momentum in research and advocacy. First, we explore the groundbreaking personalized treatment advances led by the n-Lorem Foundation. Although this therapy, which is still in development, is tailored to individuals who have a specific intronic mutation in the GBE1 gene, the implications are big. An “n-of-1” model, developing individualized therapies based on a patient’s unique genetic profile, opens the door to a new era in APBD care.

Why Raising Funds for APBD Research Matters Now More Than Ever. We are excited to launch the 2025 APBD Tour de Friends Rally for Research fundraising campaign, aiming to raise $150,000.

The APBD Research Foundation is happy to team up with two UK-based organizations, Alex - The Leukodystrophy Charity (Alex TLC) and the Association for Glycogen Storage Disease-UK (AGSD-UK), to host a special International APBD Patient Chat.

Raising Awareness on Rare Disease Day, Innovative MRI Biomarker Study Still Needs a Few More Patients

Our 2024 Year-End Giving Campaign is a Success!, Million Dollar Bike Ride Grants Awarded for APBD Research

Natacha Pires, First Executive Director, Association for Glycogen Storage Disease, GSD IV Disease, Mother's Perspective on APBD, Circle of Sustainers, Show Our Stripes, Rare Disease Day