Click here to read the Report. Check out pages 16-17 to read APBDRF's key achievements during the grant. We are excited to share the recently released Chan Zuckerberg Initiative (CZI) Rare As One Impact Report. [Read More]
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. Read Melanie’s narrative Melanie shared, [Read More]
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 [Read More]
We are excited to share that a powerful narrative from OB/GYN Dr. Larry Arky who was diagnosed with APBD in April of 2023, has been published on KevinMD.com. Read Dr. [Read More]
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.” Becca Reef, MS, CGC, our Scientific Coordinator, spoke on [Read More]
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 [Read More]
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 [Read More]
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. With Costello Medical’s support, we will develop a comprehensive [Read More]
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. Growing up just outside of Boston, Massachusetts, Becca was inspired to focus on making the [Read More]
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. We are honored to [Read More]
We are excited to share that we are hosting our virtual APBD Scientific and Community Conference on September 18-19, 2024. Join us as we bring together stakeholders in the APBD community – researchers, health care providers, industry partners, and individuals and [Read More]
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. Together, we soared to new heights [Read More]
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. [Read More]
Kerry Wong 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. The stories featured in this book are firsthand narratives from individuals [Read More]
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. Leading up to the day, we urged our community to wear [Read More]
The APBD Research Foundation is pleased to share that Or Kakhlon, PhD (Research Fund of the Hadassah Medical Organization) and Priya Kishnani, MD, MBBS and Rebecca Koch, PhD, RDN, LDN (Duke University Medical Center) are the recipients of rare disease [Read More]
We are excited to share that 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. Golden Heart [Read More]
The APBD Foundation is excited to announce key additions to its Scientific and Medical Advisory Board (SMAB). From left to right: Jennifer Orthmann-Murphy, MD, PhD; Felix Nitschke, PhD; Matthew Gentry, PhD; and Wyatt Yue, PhD [Read More]
The APBD Research Foundation is delighted to introduce our newest team member, Anesa Kajtazovic, who has joined us as our part-time Community Affairs Officer. Anesa brings with her a wealth of experience from the realms of nonprofits and governmental [Read More]
New York, NY – The APBD Research Foundation is excited to contribute to the Glycogen Storage Disease (GSD) Value Project. This initiative will develop an internationally applicable set of patient health outcomes that are relevant to individuals with liver GSDs [Read More]