02/26/2026
We're honored to collaborate with Finding Hope for Frizzle - Frrs1l to advance a lifesaving gene therapy for children with Frizzle (FRRS1L) disease.
This parent-led nonprofit has selected Parexel to provide regulatory and FDA Investigational New Drug (IND) support, as they work toward their goal of beginning clinical trials by September 2026.
FRRS1L is a devastating rare genetic disease that causes early-onset epilepsy, hyperkinetic movements, developmental delays and severe regression by age two. As we approach on Feb. 28, we're reminded of the urgency and importance of advancing treatments for the 100+ known Frizzle patients worldwide ─ and the 300-400 million people globally living with rare diseases who are fighting for treatments.
Learn more about the work that's helping to bring hope to families affected by FRRS1L. 🔗https://brnw.ch/21x0hv3