Table of Contents

  • Year-End Giving: New Match Opportunity
  • Groundbreaking APBD Research Updates
  • Dr. Or Kakhlon Explains Potential Cocktail Approach to APBD Therapies
  • Dr. Priya Kishnani and Dr. Rebecca Koch Explain How Patients' Tissues Offer Insights into Disease Progression
  • Dr. Larry Arky’s APBD Journey Reaches Millions
  • Patient Perspectives Shared with the FDA
  • In Memory of Those We Lost in 2024
  • Upcoming Events
  • Create Your Legacy with FreeWill


Wishing you and your family a joyous and peaceful Holiday Season!

Editor's Note: This oil pastel artwork was created by patient Alma Hecht. Our thanks to the Hecht family for sharing it with our community.



JUST IN – Second Match Challenge to Boost Our Year-End Fundraising Campaign

We’re thrilled to announce that Ora Gordon, MD, MS, FACMGG, has generously offered a new $25,000 gift matching opportunity for our APBD community! 


This match offer builds on our current $80,000 Board match challenge. Now through December 31st, your year-end gift to the APBD Research Foundation will have triple the impact.

Make Your Year-End Gift Today

P.S. If you have already made a year-end gift, thank you for your generosity!

 

Your generosity sustains the Foundation’s life-changing work. Thank you in advance for choosing to support the APBD Research Foundation! 

Groundbreaking APBD Research Updates

Dr. Or Kakhlon Explains Potential Cocktail Approach to APBD Therapies

Dr. Or Kakhlon shares promising insights into a potential “cocktail” approach to APBD therapy. This strategy builds on decades of research, bringing us closer to more effective treatments.

Read the Q&A

Dr. Priya Kishnani and Dr. Rebecca Koch Explain How Patients' Tissues Offer Insights into Disease Progression

Duke University’s natural history study and biospecimen research are key to improving future APBD clinical trials. This research will enhance our understanding of disease progression and help refine trial design.

Read the Q&A

Dr. Larry Arky’s APBD Journey Reaches Millions



We are excited to share that a powerful narrative from Larry Arky, MD -- an OB/GYN who was recently diagnosed with APBD -- has been published on KevinMD.com.


The story offers Dr. Arky's perspective on APBD, raising awareness about this ultra-rare neurogenetic condition and the often long, uncertain diagnostic journey that many individuals face.

Read more

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.


Becca Reef, MS, CGC, our Scientific Coordinator, spoke on behalf of the APBD community at the 2:11:36 timestamp, sharing insights on the challenges of trial enrollment during early stages of disease progression.


👉 Watch Becca’s 4-Minute Presentation here

👉 Access the Summary Slide here

👉 Add your own perspective to the open FDA docket here (through 02/03/2025)

In Memory of Those We Lost in 2024

It is with heavy hearts that we acknowledge the individuals in our community who passed away this year. We honor their beauty and their strength. Our thoughts are with their family members and friends.

Upcoming Events

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.

Patient Chats

Jan. 22, 2025, 8pm ET | 5pm PT

Feb. 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

Jan.9, 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.

Create Your Legacy with FreeWill

In just 20 minutes, you can create a will that secures your family’s future and supports APBD research.

Start my Legacy

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.

We provide this FREE monthly newsletter resource to all of our community members.

This program is made possible by contributions to the APBD Research Foundation from donors like you.

Thank You!


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