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2025


July 2025 • New addition to our team, updated prevalence estimates…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

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

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