Ohio Sanfilippo mom shares her son’s experience participating in a clinical trial

An Ohio mom shares her story and talks about her son Oliver’s journey from his Sanfilippo Syndrome (type B) diagnosis at age 4, to his participation in a gene therapy clinical trial at age 5, and an update on how Oliver’s doing today.

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Jennifer’s son Oliver was diagnosed with Sanfilippo Syndrome type B at the age of 4.  By age 5, Oliver was fortunate enough to be able to participate in a gene therapy clinical trial (specific to Sanfilippo type B) that was taking place a mere 4 miles from their home in Columbus, Ohio. This was at the height of COVID, mind you, so that added convenience of being so close by to the facility was perhaps the pivotal factor in Oliver’s participation. She describes Oliver as a “destructively happy kid” - which obviously made the process of actually getting accepted into the trial a particularly arduous and mentally/physically exhausting experience at the time.  In the following brief (3 minute) clip, Jennifer introduces herself and shares her family’s experience in navigating the terrain of their new Sanfilippo world from diagnosis until now. ​