APBD Research Foundation

02/19/2026

We’re excited to share that Ami Magazine featured an insightful personal narrative from Bina T.’s APBD journey last month. Bina’s story speaks to the long road so many face before receiving a rare disease diagnosis and the strength she found through her faith.

Bina shared, “In 2015, when I was in my mid-50s, I started having some issues with bladder control, which was a bit unsettling. The doctor told me it’s not uncommon for women who’ve had a bunch of children to suffer from it, and it could also be a function of aging. I did kegel exercises, and, for the most part, just learned to live with a dysfunctional bladder. I didn’t realize that it was a marker of something more ominous.”

Read the article here: https://loom.ly/BQBSHSY
(Ami Magazine - Ami Living Supplement, Issue 751, Jan. 14, 2026, page 42)

02/18/2026

⏰ The countdown is on: just 10 days until Rare Disease Day!

There’s still time to get involved! Join a local event, share your story online, or light up your home/office to show your support for the rare disease community!

🌍 This global day is about awareness, solidarity, and action. Every post shared, every event attended, and every light that shines helps make the rare visible and reminds us that we are stronger together.

Learn more about how to participate, find events near you, or access campaign materials: https://go.rarediseaseday.org/RDD

Let’s make this year’s Rare Disease Day more than you can imagine. 💪

02/17/2026

Be part of the research shaping the future of APBD.

Next week on Wednesday, February 25 at 9am PT / 12pm ET, join a special Patient Chat with Lindsay Gill, PhD, Research Manager at the APBD Research Foundation, to learn why participating in the GSD IV Biobank is so critical to advancing research.

📩 Email Harriet at harriet@apbdrf.org to learn more or register.

02/16/2026

♥️ Random Acts of Kindness Week (Feb. 15–21)

One of the kindest things you can do—for yourself, your loved ones, and the APBD community—is to plan ahead.

We’ve partnered with FreeWill to help you plan your legacy in just 20 minutes, at no cost—ensuring the people and causes you care about most are protected.

Care for what matters most today and for the future.

🔗 Get started: https://loom.ly/qJFz3Vc

02/12/2026

Throwback Thursday! Advancing APBD Awareness & Clinical Momentum

Golden Heart Flower (GHF) was one of five start-up finalists highlighted at the Israeli American Council - IAC National Summit's Tech Session.

The IAC National Summit — held in Hollywood, Florida on Jan. 15-17, 2026 — drew over 4,000 participants from across the Israeli-American community. Among those in attendance were representatives from the APBDRF (Emil Weiss, co-founder and co-president) and Israel-based APBD patient organization MIFNEH (Uri Gabay, a volunteer and the son of a person living with APBD). Both supported the APBD awareness-building efforts.

The GHF team took the opportunity to bring visibility to APBD at a crucial time as they work to bring GHF-201 into clinical trials. GHF-201 is a small molecule therapy designed to reduce polyglucosan body accumulation — the underlying cause of key symptoms, including bladder dysfunction and progressive loss of mobility — in APBD patients.

Read more: https://loom.ly/WCpEjjw

02/10/2026

Want to know how your participation can help move APBD research forward?

Join us for a special Patient Chat on February 25 | 9am PT / 12pm ET, featuring our Foundation’s Research Manager Lindsay Gill, PhD. Lindsay will share why participation in the GSD IV Biobank is so important—and how it can help advance research for our community.

📩 To learn more or register, email Harriet at harriet@apbdrf.org.

02/09/2026

APBD Research Update!

University of Texas Southwestern Medical Center researchers Dr. Felix Nitschke and Dr. Mayank Verma -- along with Johns Hopkins/Kennedy Kreiger researcher Dr. Nirbhay Yadav -- share updates on their collaborative work to track disease changes more accurately. They are measuring and comparing several biological markers, including MRI scans that show glycogen buildup, to better understand how the disease progresses and to help prepare for future clinical trials.

Read the Q&A: https://loom.ly/VSrZ148

02/09/2026

Did you know rare diseases affect 300 million people worldwide?

📊 Rare diseases affect 5% of the global population, that’s over 300 million people. Yet access to diagnosis, care, and treatment still depends on where you live.

Around the world, disparities in healthcare systems, specialist availability, and coverage mean that too many people with a rare disease go without the care they need.

⚖️ Equality would mean offering everyone the same system.
💜 Equity for rare diseases means acknowledging that not everyone starts from the same place, and committing to policies and investments that close the gap.

👉 Learn more about equity: https://go.rarediseaseday.org/equity

02/08/2026

IN CASE YOU MISSED IT! Here's our January newsletter: https://loom.ly/GehJcWk

Check out what’s inside:
- Our 2025 Year-End Giving Campaign is a success
- Honoring former board member David Epstein
- Groundbreaking research updates
- Dr. Wyatt Yue explains how targeting other steps of glycogen production can treat APBD
- Dr. Felix Nitschke, Dr. Nirbhay Yadav, and Dr. Mayank Verma explain the importance of developing biomarkers for APBD
- Share your voice: Take our GSD IV BioBank survey
- Golden Heart Flower raises APBD awareness
- APBD spotlighted in Ami Magazine
- Upcoming Chat events for patients and caregivers

02/06/2026

💙 Help build our GSD IV BioBank.

YOU can now help us accelerate biomarker discovery and drug development for all forms of GSD IV! Join our newly launched GSD IV Biobank -- a vital tool for our research community. Take the first step with our interest survey: https://loom.ly/OuYr_rI

02/04/2026

APBD Research Update!

Dr. Wyatt Yue (Newcastle University) describes a treatment approach for APBD that uses small molecules to target glycogen synthase, a protein that helps make glycogen. This protein works earlier in the process than the glycogen branching enzyme. By using this approach, the treatment aims to reduce the buildup of abnormal glycogen, called polyglucosan. This strategy is gaining interest among researchers studying APBD and related diseases, including Lafora disease and Pompe disease.

Read the Q&A: https://loom.ly/p840imw

02/03/2026

Join us for a special Patient Chat with a guest speaker on February 25th, 9am PT | 12pm ET. Lindsay Gill, PhD, Research Manager with the APBD Research Foundation, will be presenting on Why It's Important That You Participate in the GSD IV Biobank.

To learn more or register for this event, email Harriet at harriet@apbdrf.org.