10/08/2026
We're so excited and thankful for the opportunity to be representing the APBD community at the upcoming National Organization for Rare Disorders, Inc. (NORD) Breakthrough Summit later this month. Lindsay Gill, PhD, our Research Director, will be sharing the findings of our "Clinical Trial Readiness Survey" during the poster session. She has also been selected to share emerging findings and new perspectives during Lightning Round Presentations! 🫶
We wish to thank our APBDRF board, staff, genetic counseling intern Dylan Ross (Columbia University), and Scientific and Medical Advisory Board Co-Chairs Dr. Jennifer Orthmann-Murphy (UPenn) and Dr. Felix Nitschke (UTSW Medical Center) for their help with developing the survey and the outreach efforts. Also, we want to acknowledge Drs. Michio Hirano (Columbia University), Niccolo Mencacci (Northwestern University), and Eric Mallack (Kennedy Krieger Institute), and Dr. Rebecca Koch (Duke University) for helping review the survey and the data, and for supporting the recruitment efforts. Most important, we are thankful to our community members for adding their voice to this survey!
Rare disease research takes the spotlight at the 2026 ⚡️
Five researchers among many on view will share their work during Lightning Round Poster Presentations of research advancing rare disease care.
Join us Oct. 26–27 in Washington, D.C., or virtually to hear these researchers present their work: https://bit.ly/4jPFNwe
Featuring representatives from: Merck, Emily Whitehead Foundation, APBD Research Foundation, Howard University College of Medicine and Children's National - Main Hospital, and Parent Project Muscular Dystrophy.
