Symptoms of PoTS You Shouldn't Ignore

Understanding Tourette Syndrome: Gisela's Journey

This video captures just over one month in the lives of families fighting Sanfilippo syndrome type B — a rare, terminal childhood disease. In less than 36 days, families and communities across the country raised $4.2 million by Thanksgiving, and met our $5.5 million goal by December 1, to pursue potential early access to a treatment many parents were never told existed. That milestone matters. But it is not the finish line. The process still in front of us — access decisions, timelines, regulato