The Oct. 27, 2021, episode of the Foundation’s “A Close Look at Sanfilippo” podcast spends 10 minutes (and a little more in this episode) diving into 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, shares footage from his presentation during an EveryLife Foundation scientific workshop in which he made the case for why Sanfilippo Syndrome is an ideal candidate for the FDA’s accelerated approval program and how there are likely multiple treatments that could start helping children with Sanfilippo in some way right now. “It’s urgent to us! The children [with Sanfilippo] are getting worse every second as the storage of heparan sulfate accumulates in every cell. Sanfilippo is not a disease to be conservative regarding the drug development path … Accelerated approval could give these children a chance at better days, more time, and maybe more comfort. Let’s start with that, and we can improve on it from there. As one of our parents stated, ‘Doing nothing is the greatest risk in Sanfilippo.’”
Katie Walton, VP of Marketing for the Foundation, introduces a new resource created for parents of children newly diagnosed with Sanfilippo. The brief guide walks through initial questions most families have about Sanfilippo and what to do next, to help them proactively care for their child and give them the best quality of life.
Next Glenn dives into how donors’ generous support of the Foundation has made more than 30 research projects possible in just eight years, some of which have moved from promising lab research into clinical trials. Plus, how the Foundation’s diverse approach to funding research — exploring multiple treatment and therapeutic options, supporting programs for early detection of Sanfilippo (physician education, newborn screening), and creating of global clinical management guidelines for Sanfilippo — all contribute to improving the lives of children with Sanfilippo.
For the final two topics in this episode, Glenn shares how people can participate in the third annual World Sanfilippo Awareness Day on Nov. 16 and the end-of-year goals for the Foundation and how people’s generous hearts make reaching $2 million in fundraising each year a possibility. “We’re so thankful. … In this podcast we showed you what impact the dollars for research lead to. The impact and the difference donors are making is clear.”
A Close Look At Sanfilippo” is a regular 10-minute podcast from Cure Sanfilippo Foundation. Each episode spotlights five hot topics in the pursuit of finding a treatment and giving children with Sanfilippo more good days. And how the Foundation is involved and how it accelerates the mission to get an FDA-approved treatment for the children.
Each “A Close Look At Sanfilippo” podcast is a way for partner families, donors and supporters, researchers and clinicians, and industry partners to hear directly from us. One that you can listen to at your convenience.