On #WorldSanfilippoDay we honor those affected by Sanfilippo syndrome—a rare disease known as childhood Alzheimer’s where a genetic defect leads to buildup of toxins in the brain & early death. rareLife is dedicated to advancing treatments & improving outcomes for the rare community.
#WeKnowRare

You've probably never heard of it It's a rare disease, after all. And terminal.

My first hashtag here is also its first instance on Bluesky: #WorldSanfilippoDay (11/16)

You’ve probably never heard of it. It’s a rare disease. And terminal. Like Alzheimer’s—but in kids. Affects 1/70k births. Including my nephew:

www.linkedin.com/pulse/youve-...

curesanfilippofoundation.org