October 2026 • Vision Loss Focus Group, Biomarker Research Opportunities…and more!
UCLA researchers Drs. Gal Bitan and Brent Fogel are now accepting participants -- individuals who have APBD or other forms of GSD IV, as well as healthy participants -- at NYU (New York, NY) in addition to UCLA (Los Angeles, CA) for their biomarker study. Their work examines blood-based biomarkers that may point to changes in the brain. Participation involves an in-person blood draw and completing health questionnaires and assessments.
August 2026 • TAKE ACTION: Shape Research with Your Lived Experience
Sharing your experience to inform research is crucial as we work to advance therapeutic development efforts. Whether you're a patient, a caregiver or family member, or a healthcare professional, here are some opportunities for you
May 2026 • Gene therapy research update; trivia event; and more!
The APBD Research Foundation has awarded its second and third 2025 Rally for Research pilot grants to UTSW Medical Center researcher Dr. Mayank Verma and Duke University researcher Dr. Rebecca Koch.
April 2026 • 34,000+ reasons to make treatments a reality
The APBD Research Foundation is excited to share that its global genetic prevalence* of GBE1-related diseases* study has been published in the Journal of Inherited Metabolic Disease Reports (JIMD Reports).
March 2026 • Join new APBD biomarker study + major research updates
UCLA researchers Gal Bitan, PhD and Brent Fogel, MD, PhD are seeking volunteers for their research study that aims to identify blood-based biomarkers* which point to changes in the brain for people who have APBD and other forms of GSD IV.
February 2026 • Biomarker research news, n-of-1 trial milestone…and more
2025 Rally for Research Series: UCLA Scientists Awarded Pilot Grant; n-Lorem Foundation Receives FDA Authorization to Proceed with n-of-1 Trials for ASO Therapy..
January 2026 • Foundation mourns the passing of former board member David Epstein; research updates
Our 2025 Year-End Giving Campaign is a success; Honoring former board member David Epstein; Groundbreaking research updates
December 2025 Newsletter — 34,000 genetic prevalence of GSD IV preprint; Dr. Matthew Gentry receives Rare Disease Scholar Award
34,000 genetic prevalence of GSD IV preprint; Dr. Matthew Gentry receives Rare Disease Scholar Award
November 2025 Newsletter — GSD IV Biobank launch; how to create your CRID
Your support and belief in our mission sustains our work on behalf of more than 34,000 people living with APBD and related diseases. Thanks to you, we are making real progress toward new treatments that are moving closer to fruition
October 2025 Newsletter — Participate in the “Living Rare Study,” Carol Rosenstock shares her APBD journey…
While rare diseases impact millions, there is very little recognition of the severity of the public health impacts that they generate. Why is that? Because relatively little data has been developed to document the extraordinary breadth of the impacts.
September 2025 Newsletter — Foundation mourns the passing of co-founder Michael Weiss; research highlights…
The APBD Research Foundation is deeply saddened to share that our co-founder, Michael Weiss, passed away on September 6, 2025, due to complications from APBD.
July 2025 Newsletter — New addition to our team, updated prevalence estimates…and more!
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.
May 2025 Newsletter — Progress with a noteworthy therapy; presymptomatic diagnoses;…and more!
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.
April 2025 Newsletter —Rallying for Research, COMBINEDBrain partnership…and more!
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.
March 2025 Newsletter —International Patient Chat; Research Highlights; … and more!
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.
February 2025 Newsletter — Recognizing Rare Disease Day; biomarker studies; and more!; …
Raising Awareness on Rare Disease Day, Innovative MRI Biomarker Study Still Needs a Few More Patients
January 2025 Newsletter — APBD research grants awarded; join our Rare Disease Day efforts; …
Our 2024 Year-End Giving Campaign is a Success!, Million Dollar Bike Ride Grants Awarded for APBD Research
February 2023 Newsletter — Rare Disease Day; a Mother’s Perspective…and more!
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