Everyday giving to support lives of children with Sanfilippo Syndrome image

Everyday giving to support lives of children with Sanfilippo Syndrome

We are working on our Ambroxol trial for all types and ages of Sanfilippo patients.

$63,101 raised

$500,000 goal

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Giving quality of life to children with Sanfilippo Syndrome!

  1. Our Ambroxol trial is currently underway in Virginia. If approved, this treatment will be effective for all types, and all ages of Sanfilippo patients. As always, we will continue to research quality of life treatments for our entire community.