The FDA delayed a gene therapy that could save children with Sanfilippo syndrome — a disease that steals children’s voices, mobility, and memories.
Please sign the letter now. Add your voice to the 25,000 we have.

🔗 tinyurl.com/UNC-RareDis

#ApproveHopeNow #WalkTheTalkMakary #SanfilippoSyndrome

Veda's favorite animal is a turtle 🐢 My daughter has Sanfilippo syndrome, and "turtle" is one of the few words she's still able to say. #Sanfilippo #sanfilliposyndrome #veda #mps #MPSIII

My daughter Veda has Sanfilippo syndrome. It's a rare, terminal illness that currently has no FDA approved treatment. We are busy raising awareness and fundraising for a cure. 💜