APBD Research Foundation

📣Calling all APBD and early-onset GSD IV patients and caregivers!

✍️The APBD Research Foundation is conducting a 10-15 minute, anonymous survey of patients and caregivers to prepare our community for future clinical trials -- referred to as “clinical trial readiness.” Your participation in this survey will help us understand what matters to patients and their families when it comes to participating in clinical trials for new treatments.

✅Check out our recruitment page for more details and to access the surveys 👉 https://loom.ly/MElvLoQ
⏰NOTE: Surveys will be open until October 1, 2026.

Happy Independence Day! 🇺🇸

Wishing you a safe, joyful, and beautiful Fourth of July weekend!

💜 Thanks to the generosity of our community during last year's Rally for Research campaign, the APBD Research Foundation recently funded three pilot research grants:

- Gene Therapy to Treat the Neurophenotype of Glycogen Storage Disease Type IV | Dr. Rebecca Koch - Duke University

- Preclinical Validation and Biomarkers in APBD of a GYS1-ASO Currently in Phase 1 Clinical Trial for Lafora Disease | Dr. Mayank Verma - UTSW Medical Center

- New Biomarkers for APBD | Dr. Gal Bitan and Dr. Brent Fogel - UCLA

Read about each of these research initiatives by visiting: apbdrf.org/research-highlights/research-grant-announcement