
08/04/2025
✅ ACTION ALERT: Help save kids in only 20 seconds.
🧬 Children with Sanfilippo syndrome and other rare diseases don’t have time to wait.
The FDA has rejected a promising gene therapy, despite admitting that the clinical data is strong. Now, families may have to wait a year or more while their children continue to decline. But momentum is shifting at the FDA and we have a small window to speak up.
📢 Sign this urgent letter to the FDA asking for a faster review and a meeting with families:
https://unc.az1.qualtrics.com/jfe/form/SV_0ivKzumYy787guq
Deadline: August 6
Your voice matters. Please sign and share.