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Table of Contents
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Our 2024 Year-End Giving Campaign is a Success!
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Million Dollar Bike Ride Grants Awarded for APBD Research
- Innovative MRI Biomarker Study Still Needs Three More Patients
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CZI's Report Highlights Our Impact in Advancing Rare Disease Research
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Join Our Rare Disease Day Efforts!
- Take Our Two-Minute Survey
- Upcoming Patient and Caregiver Chats
- Plan Your Legacy in 2025
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Thank You for Making Our 2024 Year-End Giving Campaign a Success! | |
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Dear Friend,
Our 2024 Year-End Giving Campaign was a roaring success with 151 generous donors joining us, collectively contributing $218,000 to further our mission.
With your support, we exceeded our 2024 year-end goal of $160,000. Your time, skills, donations, connections, APBD stories, and peer-to-peer campaigns help us build momentum as we work to make APBD treatments a reality. We are excited about all that you will make possible in 2025!
Thank you for standing with us and for being a vital part of our community,
The APBD Research Foundation Team
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UT Southwestern Medical Center and Newcastle University Scientists Awarded Million Dollar Bike Ride Grants for APBD Research | |
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The APBD Research Foundation is thrilled to announce that Felix Nitschke, PhD (UT Southwestern Medical Center) and Wyatt Yue, PhD (Newcastle University), have been awarded rare disease research grants through the University of Pennsylvania’s Million Dollar Bike Ride (MDBR) program. Dr. Nitschke and Dr. Yue, both past recipients of MDBR grants, are eager to expand on the foundation of their previous work and drive it forward.
Dr. Nitschke, in collaboration with co-investigators Mayank Verma, MD, PhD (UT Southwestern) and Nirbhay Yadav, PhD (Johns Hopkins University), will focus on developing reliable imaging, urine, and blood biomarkers to monitor disease progression and assess the effectiveness of potential treatments in future clinical trials.
Dr. Yue will build on the progress enabled by the MDBR grant he received in 2021. His team aims to create a groundbreaking oral therapy by combining an innovative screening approach with their exceptional chemical and biochemical expertise.
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Since we started partnering with the UPenn's MDBR program in 2016, our researchers at Columbia University, Duke University Medical Center, UT Southwestern Medical Center, University of Florida, Newcastle University, and Hadassah Medical Organization, have received $873,000 in pilot grants to advance APBD research.
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Research Highlight: Help Advance APBD Biomarkers Research Through an Innovative MRI Study
We need at least three more volunteers to participate in a groundbreaking APBD biomarker study led by Dr. Nirbhay Yadav (Johns Hopkins University).
Dr. Yadav is pioneering new MRI methods to image glycogen itself. If successful, these images could serve as critical disease biomarkers, paving the way for future clinical trials.
If you are able to travel to Baltimore (MD) to participate in this study, here is more information:
Location: Kennedy Krieger Institute
Address: 707 N. Broadway, Baltimore, MD 21205 (Johns Hopkins Medical Campus)
Volunteer Requirements: Adults diagnosed with APBD are needed to undergo magnetic resonance imaging (MRI) scans of the brain or body. This research is being done to develop new types of MRI scans that are more sensitive to tissue changes compared to current methods. Volunteer participants are required to lay within the MRI scanner for up to 60 minutes. The MRI scan does not involve any radiation or injections.
For more information, please email Nirbhay Yadav, PhD at nyadav@jhu.edu.
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CZI’s Cycle 1 Rare As One Impact Report Features APBDRF’s Work, Alongside Peers, to Advance Rare Disease Research | |
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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. Check out pages 16-17 to read APBDRF's key achievements during the grant.
CZI has since expanded the Rare As One Network to include 94 patient-led rare disease organizations. Although our funding cycle concluded in 2022, our Foundation continues to be an active participant of the RAO network.
As APBDRF co-president Jeff Levenson, DDS shared in the report, “Our biggest gains came from connections made during our Scientific and Community Conferences and our Biomarkers Workshops, as well as [collaborative research grant] applications. The RAO grant funding, educational programs, and expert support, along with the camaraderie of the other grantees, also helped us focus on what are now promising therapeutic initiatives.”
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Join Our Rare Disease Day Efforts!
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We have an exciting plan for Rare Disease Day, February 28, and we invite you to be part of it!
A Foundation supporter who believes in spreading awareness donated some special "wearables" to enhance Rare Disease Day selfie portraits of our community members. We have zebra-striped pins along with zebra-patterned ribbon to help you reflect the day's theme, Show Your Stripes. The pins and ribbon can be used together or separately—creativity is encouraged!
Upon request, we’ll ship the wearables to you. In return, we ask for a selfie where you show your stripes. Photos may be included in our Foundation’s awareness campaign.
This unique opportunity is available on a first come, first served basis for all APBD patients, family members, friends, researchers, and clinicians.
Don’t miss out! Email Faye Rosenberg at feigymrosenberg@gmail.com to request your pins and ribbon today! Selfies are due back to Faye by February 10.
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Take Our 2-Minute Survey and Let Us Know What You Think! | |
We want to hear from you! Take our brief survey to share your thoughts and help us communicate with, and serve, you better. Your voice matters. | |
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From Chat events to workshops and scientific symposiums, the Foundation hosts programs for patients, caregivers, and health professionals which connect our community members and deepen the understanding of how APBD impacts lives.
These events offer peer support, opportunities to hear from health experts and Foundation leaders, and the chance to raise funds for APBD research.
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Patient Chats
Feb. 26, 2025, 8pm ET | 5pm PT
Mar. 26, 2025 8pm ET | 5pm PT
The Patient Chat is hosted by volunteer moderator Harriet Saxe. To learn more, email Harriet at harriet@apbdrf.org.
Caregiver / Family Chats
Feb. 13, 2025 7:30pm ET | 4:30pm PT
Mar. 13, 2025 7:30pm ET | 4:30pm PT
This Chat is hosted by volunteer moderator Linda Cedarbaum. To learn more, email Linda at linda@apbdrf.org.
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As we step into the new year, now is the perfect time to plan your legacy. Creating a will has never been easier or more impactful! In as little as 20 minutes, you can secure your family's future and support life-changing APBD research.
The APBD Research Foundation partners with FreeWill, a trusted tool used by over a million people. With your will, you can outline your wishes, bring peace of mind to your loved ones, and leave a lasting legacy of hope.
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If you have any questions about FreeWill or legacy giving, please contact Anesa Kajtazovic at anesa@apbdrf.org.
P.S.: If you have already included a gift to the APBD Research Foundation in your estate plan, please fill out this form! We’d love to thank you for your generosity.
Disclaimer: Please note that FreeWill is not a law firm and cannot provide legal advice, nor can the APBD Research Foundation. We would always encourage that a lawyer review your will and any other important legal documents FreeWill may generate for you.
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We provide this FREE monthly newsletter resource to all of our community members.
This resource is made possible by contributions to the APBD Research Foundation from donors like you.
Thank You!
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