Foundation Partners with Alex TLC and AGSD-UK to Host a Special Patient Chat Aimed at Engaging the International Community
The APBD Research Foundation is excited to partner with UK-based Alex The Leukodystrophy Charity (Alex TLC) and Association for Glycogen Storage Disease – UK (AGSD-UK) on a special APBD Patient Chat that aims to engage the global community.
APBD Community Joins Millions of Rare Advocates Around the World for Rare Disease Day
The APBD Research Foundation rallied its community to mark Rare Disease Day on February 28, joining the National Organization for Rare Disorders and rare disease advocates around the world.
CZI’s Cycle 1 Rare As One Impact Report Features APBDRF’s Work, Alongside Peers, to Advance Rare Disease Research
We are excited to share the recently released Chan Zuckerberg Initiative (CZI) Rare As One Impact Report. The report highlights the accomplishments of our Foundation and 29 other Cycle One rare disease organizations over a three-year grant period that ended in 2022.
Melanie Robinson’s Caregiving Journey Reaches Millions
We are excited to share that Melanie Robinson’s narrative on caregiving for her mother, Ginny, who was diagnosed with APBD in 2018, has been published on KevinMD.com.
University of Texas Southwestern Medical Center and Newcastle University Scientists Awarded Million Dollar Bike Ride Grants for APBD Research
The APBD Research Foundation is pleased to share that Felix Nitschke, PhD (University of Texas Southwestern Medical Center) and Wyatt Yue, PhD (Newcastle University) are the recipients of rare disease research grants from the University of Pennsylvania’s Million Dollar Bike Ride (MDBR) program.
Dr. Larry Arky’s APBD Journey Reaches Millions
As an OB/GYN for the past 22 years, I have loved delivering babies, performing surgeries, and having long-term relationships with my patients. Now, I am on the other side of things, as a patient myself. I was diagnosed with adult polyglucosan body disease (APBD) in April of 2023 at the age of 59.
Patient Perspectives Shared with the FDA
The APBD Research Foundation recently participated in the FDA’s Listening Session on “Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases.”
Foundation on the Road: Advocating for You and Learning from Peers at Key Stakeholder Meetings
On the heels of our recent APBD Scientific & Community Conference, the Foundation team represented our community in person and virtually at key stakeholder convenings across the country. At the cornerstone of our work is the belief in advocacy and collaboration. We believe that through these efforts, we can advance APBD research and drug development.
2024 Virtual APBD Scientific & Community Conference is a Success!
On September 18-19, over 200 attendees joined the 2024 APBD Scientific & Community Conference. The virtual event brought together stakeholders in the APBD community – researchers, health care providers, industry partners, and individuals and family members impacted by APBD.
APBD Research Foundation Receives Crucial Support from Costello Medical to Build Bridges Between the APBD and GSD IV Communities
The APBD Research Foundation is excited to announce a new collaboration with Costello Medical, Inc., following the Foundation’s successful application to Costello Medical’s pro bono project initiative for Rare Disease organizations.
Meet Becca Reef, Our Newest Team Member!
The APBD Research Foundation is delighted to introduce our newest team member, Becca Reef, MS, who has joined us as our part-time Science Coordinator.
APBD Research Funding Opportunity Through UPenn’s Million Dollar Bike Ride Pilot Grant Program
The APBD Research Foundation, in partnership with the Orphan Disease Center (ODC) at the University of Pennsylvania, is pleased to announce the launch of the 2024 Million Dollar Bike Ride (MDBR) Pilot Grant Program.
Foundation to Host APBD Scientific and Community Conference
We are excited to share that we are hosting our virtual APBD Scientific and Community Conference on September 18-19, 2024.
APBD Tour de Friends Team Raises a Record-Breaking $134,000 During the 2024 Million Dollar Bike Ride For Rare Disease Research
On June 8th, our APBD Tour de Friends team gathered on the ground in Philadelphia, biking, walking, and scootering, while over 271 virtual participants and donors joined in support from across the country. Our community set a new fundraising record this year, reaching $134,742.
APBD Research Foundation’s Focus Group Brings APBD and GSD IV Communities Together for the First Time
On May 6, 2024, the APBD Research Foundation hosted a virtual Focus Group that brought together, for the first time, over 50 participants representing the Adult Polyglucosan Body Disease (APBD) and Glycogen Storage Disease Type IV (GSD IV) communities.
APBD Featured in New Book, Kaleidoscope: Rare Disease Stories
We are excited to share that Sarah William’s APBD journey is featured in the newly published book, Kaleidoscope: Rare Disease Stories, by Kerry Wong.
APBD Community Shines Bright on Rare Disease Day
The APBD community joined the National Organization for Rare Disorders and rare disease communities around the world to celebrate Rare Disease Day on February 29th.
Duke University and Research Fund of the Hadassah Medical Organization Scientists Awarded Million Dollar Bike Ride Grants for APBD Research
The APBD Research Foundation is pleased to share that Or Kakhlon, PhD and Priya Kishnani, MD, MBBS and Rebecca Koch, PhD, RDN, LDN are the recipients of rare disease research grants from the University of Pennsylvania’s Million Dollar Bike Ride program.
Golden Heart Flower Granted FDA Orphan Drug Designation for GHF-201 for the treatment of Glycogen Storage Diseases Type IV, including APBD
Golden Heart Flower, Ltd. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s product GHF-201 to treat glycogen storage disease type IV, including APBD.
APBD Research Foundation Expands its Scientific and Medical Advisory Board with the Addition of Dr. Jennifer Orthmann-Murphy, Dr. Felix Nitschke, Dr. Matthew Gentry, and Dr. Wyatt Yue
The APBD Foundation is excited to announce key additions to its Scientific and Advisory Board (SMAB).