Chan Zuckerberg, Sanfilippo biomarkers & FDA; Anakinra trial update; + more

A Close Look at Sanfilippo

Aug 25 2021 • 10 mins

Episode 1. “A Close Look At Sanfilippo” is a regular 10-minute podcast covering five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no FDA-approved treatment.

In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about the Foundation's application for the Chan Zuckerberg Initiative Rare As One grant and how it could accelerate several global key research and collaboration projects; how use of biomarkers to approve a new Alzheimer’s drug could open doors for Sanfilippo and other degenerative diseases that can’t wait for “just perfect” options; how families of children with Sanfilippo sharing their medical records can accelerate scientists’ development of trials and treatments; the many ways people are fundraising and supporting the Foundation, enabling it to continue its mission to find a treatment or cure; and an update on the Anakinra clinical trial’s initial indicators and what comes next.

Dr. Emil Kakkis article:

For families to join AllStripes data collection:


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Music credit: Springish by Gillicuddy (
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