February 2026 • Biomarker research news, n-of-1 trial milestone…and more
Table of Contents
First 2025 Rally for Research grant awarded to UCLA researchers
FDA authorizes n-of-1 trials for ASO therapy that targets a specific APBD mutation
Growing the GSD IV BioBank
Recognizing our caregivers
New research supports view of a GSD IV continuum encompassing children and adults
Upcoming Rare Disease Day events
Upcoming Chats for patients and caregivers
Ways to support the Foundation all year long
