Newsletter Archive

Search our Newsletter Archive (beta):

2026

April 2026 • 34,000+ reasons to make treatments a reality
Table of Contents:

  • 34,000 -- new research puts a number on the global burden of APBD and other forms of GSD IV

  • Together, we can make a difference with the 2026 Rally for Research Campaign

  • Only a few days left to participate in our Drug Repurposing Study!

  • APBDRF submits commentary to the FDA on “Individualized Therapy Pathway" guidance

  • APBD research spotlighted at major neurology conference

  • Upcoming Patient Chat and Family/Caregiver Chat

March 2026 • Join new APBD biomarker study + major research updates
Table of Contents:

  • Volunteers needed for UCLA blood-based biomarker research study

  • Developing a “Disease Concept Model” for APBD

  • Carol Rosenstock joins Foundation’s Board of Directors

  • Watch Dr. Lindsay Gill discuss the “APBDRF Biobank” initiative

  • Upcoming Chats for patients and caregivers

  • Create your legacy with FreeWill

February 2026 • Biomarker research news, n-of-1 trial milestone…and more
Table of Contents:

  • First 2025 Rally for Research grant awarded to UCLA researchers

  • FDA authorizes n-of-1 trials for ASO therapy that targets a specific APBD mutation

  • Growing the GSD IV BioBank

  • Recognizing our caregivers

  • New research supports view of a GSD IV continuum encompassing children and adults 

  • Upcoming Rare Disease Day events 

  • Upcoming Chats for patients and caregivers

  • Ways to support the Foundation all year long

January 2026 • Foundation mourns the passing of former board member David Epstein; research updates
Table of Contents:

  • 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

2025

December 2025 • 34,000 genetic prevalence of GSD IV preprint; Dr. Matthew Gentry receives Rare Disease Scholar Award
Table of Contents:

  • Happy holidays

  • Unlock new match challenge opportunity

  • Pre-print published estimating the global genetic prevalence of GSD IV at 34,000

  • Dr. Matthew Gentry receives “Rare Disease Scholar Award”

  • Share your voice: take our GSD IV BioBank Survey

  • Create your Clinical Research ID (CRID)

  • In memory of those we lost in 2025 

  • Upcoming events

  • Make a tax-free impact with a gift from your IRA

November 2025 • GSD IV Biobank launch; how to create your CRID
Table of Contents:

  • Thank you for being a part of our community

  • Foundation launches GSD IV Biobank

  • Why you should create your “Clinical Research ID”

  • Give and double your impact on #GivingTuesday

  • Upcoming Chat events for patients and caregivers

  • Make a life-changing legacy gift

October 2025 • Participate in the “Living Rare Study,” Carol Rosenstock shares her APBD journey…
Table of Contents:

  • Participate in NORD’s Living Rare Study

  • Foundation's Executive Director attends NORD’s Breakthrough Summit

  • Foundation's Research Manager attends n-Lorem Foundation’s Nano-rare Patient Colloquium

  • Carol Rosenstock shares her APBD journey in the Jewish Standard

  • Laurence Letreguilly shares her family’s GSD IV journey

  • Save the Date: Dec. 2 is #GivingTuesday 

  • Upcoming Chat events for patients and caregivers

  • Make a life-changing legacy gift

September 2025 • Foundation mourns the passing of co-founder Michael Weiss; research highlights…
Table of Contents:

  • Honoring APBDRF co-founder Michael Weiss

  • ASO therapy corrects intronic variant and restores GBE activity in APBD patients’ cells

  • Dr. Lindsay Gill champions GSD IV research at COMBINEDBrain Summit

  • Henry Daumeyer joins the Foundation team as Genetic Counseling Intern

  • Robin Knoll hosts Pampered Chef fundraiser

  • Foundation launches new website

  • Upcoming Chat events for patients and caregivers

  • Make a life-changing legacy gift

July 2025 • New addition to our team, updated prevalence estimates…and more!
Table of Contents:

  • Tour de Friends Rally for Research Fundraiser Surpasses Goal

  • Welcoming Dr. Lindsay Gill, Our New Research Manager

  • Golden Heart Flower Starts Phase I Clinical Trial in Europe

  • New Estimate: 34,000 People Worldwide Could Have GBE1-Related Diseases

  • Upcoming Chats for Patients and Caregivers 

  • Create Your Legacy with FreeWill

May 2025 • Progress with a noteworthy therapy; presymptomatic diagnoses;…and more!
Table of Contents:

  • Progress with ASO therapy development for individuals with the intronic mutation

  • Navigating a presymptomatic diagnosis

  • Advancing APBD research depends on sustained funding

  • Elizabeth LeBlanc joins Foundation team as genetic counseling intern

  • Upcoming Chats for Patients and Caregivers

April 2025 • Rallying for Research, COMBINEDBrain partnership…and more!
Table of Contents:

  • Why Raising Funds for APBD Research Matters Now More Than Ever

  • Dr. Larry Arky Joins Foundation’s Board of Directors

  • Highlights from our APBD Biomarker Workshop

  • Advancing Research with COMBINEDBrain Partnership

  • Key Takeaways from Our Collaboration with Costello Medical 

  • Upcoming Chats for Patients and Caregivers

  • Create a Legacy of Hope for APBD

March 2025 • International Patient Chat; Research Highlights; … and more!
Table of Contents:

  • Connecting our Global APBD Community: A Special Patient Chat

  • Amber Robertson Shares Her Family's Journey with Childhood-Onset GSD IV

  • APBD Biomarker Workshop Engages Research Community

  • Empowering Patients: Key Takeaways from ASGCT’s Summit

  • Upcoming Patient and Caregiver Chats

  • Supporting Your #1 Cause

February 2025 • Recognizing Rare Disease Day; biomarker studies; and more!;
Table of Contents:

  • Raising Awareness on Rare Disease Day

  • Innovative MRI Biomarker Study Still Needs a Few More Patients

  • From MS to APBD--One Patient’s Diagnostic Journey

  • Understanding Carrier Screening: A Key to APBD Genetics

  • NORD’s “Rare Disease Drug Development” Resource

  • Upcoming Patient and Caregiver Chats

  • Plan Your Legacy in 2025

January 2025 — APBD research grants awarded; join our Rare Disease Day efforts;
Table of Contents:

  • Our 2024 Year-End Giving Campaign is a Success! 

  • 2024 Million Dollar Bike Ride Grants Awarded for APBD Research to Duke University and UTSW

  • Innovative Johns Hopkins University MRI Biomarker Study Still Needs Three More Patients

  • CZI's Report Highlights Our Impact in Advancing Rare Disease Research 

  • Join Our Rare Disease Day Efforts!

  • Take Our Two-Minute Survey 

  • Upcoming Patient and Caregiver Chats 

  • Plan Your Legacy in 2025

2024

December 2024 — Q&A with APBD researchers; FDA Listening Session;…and more
Table of Contents:

November 2024 — YOUR chance to be a part of research; a mother and daughter share their journey…
Table of Contents:

  • Volunteer Opportunities: Biomarker Research Studies

  • Navigating APBD: A Mother and Daughter’s Story

  • Double Your Impact on Giving Tuesday

  • Foundation Advocates at n-Lorem Patient Colloquium

  • Upcoming Caregiver & Patient Chats

  • What Will Your Legacy Be? Create Your Will Today

October 2024 — Our researchers need YOU; advocating for APBD breakthroughs…
Table of Contents:

  • Join the MRI Study to Advance APBD Biomarkers Research

  • Foundation Advocates at Key Meetings

  • APBD RF Announces Strategic Communication Initiative with Costello Medical to explore collaborations between all GBE1 disease Communities

  • Honor Caregivers with Tribute Gifts

  • Upcoming Caregiver & Patient Chats

  • Watch APBD Conference on YouTube

  • Create Your Will, Support APBD Research

September 2024 — Missed it? Catch up on our Conference highlights…
Table of Contents:

  • Conference Recap: Over 200 Participants from 15 Countries

  • Explore the Agenda and Speaker Profiles

  • Watch the Recordings You Missed

  • Key Highlights: Research, Therapeutics, and Collaboration

  • Gratitude for Our Speakers, Sponsors, and Supporters

August 2024 — Research highlights; two weeks to our virtual Conference; MDBR research funding…and more!
Table of Contents:

July 2024 — You're invited! Join us on Sept. 18-19...
Table of Contents:

  • Save the Date: APBD Scientific & Community Conference

  • Sponsorship Opportunities for the Conference

  • Uniting APBD and GSD IV Communities: Focus Group Recap

  • APBD RF Documentary “Life through a Lens” to Screen at Global Genes Event

  • Upcoming Caregiver & Patient Chats

June 2024 — Record-Breaking MDBR Campaign; Insightful Podcasts; … and more!
Table of Contents:

May 2024 — Missed it? Recording of “Swallowing & Speech Disorders in APBD” discussion…and more!
Table of Contents:

April 2024 — Why We Participate in the MDBR
Table of Contents:

  • Join Us for the Million Dollar Bike Ride on June 8

  • Impact of MDBR: Over $700,000 in Research Grants Awarded

  • Why We Ride: Stories from APBD Supporters

  • Your Gifts Matched: Double Your Impact by May 10

March 2024 — Join us for the Million Dollar Bike Ride; new rare disease book;… and more
Table of Contents:

  • Join the 2024 Million Dollar Bike Ride: Virtual & In-Person Options

  • Research Highlight: Findings from the Columbia APBD Patient/Family (CAP) Registry

  • New Book Kaleidoscope: Rare Disease Stories featured APBD’s Sarah Williams Story

  • Spotlight on APBD Research: GHF-201 Molecule

  • Upcoming Caregiver & Patient Chats

  • Leave a Legacy of Hope: Make an Estate Gift

February 2024 — Rare Disease Day; Million Dollar Bike Ride; Research Highlights;...and more!
Table of Contents:

  • Raising Awareness on Rare Disease Day

  • Research Highlight: Insights from Proteomic Investigations

  • Access GBE-1 Genetic Prevalence Estimates with GeniE

  • Welcome Alice Tran, Our New Intern

  • Upcoming Caregiver & Patient Chats

  • Save the Date: May 6 Focus Group & June 8 Bike Ride

  • Create Your Legacy with FreeWill

January 2024 — Research Highlight; Snag Your Free Sunglasses for Rare Disease Day
Table of Contents:

  • Thank You for a Record-Breaking Year-End Campaign

  • Research Highlight: Case Report Expanding APBD Understanding

  • Show Your Stripes: Free Sunglasses for Rare Disease Day

  • Welcome Matthew Morgan, Our New Intern

  • Upcoming Caregiver & Patient Chats

  • Missed December's Patient Chat? Watch It Now

2023

2022

2021

2020

2019

2018

 2017

 2016

2015

2014

2013

2008

2007

 

About Us

Summary Block
This block has no content yet. Items you add to the page connected to this block will display here.
Featured