Episode 7. “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, 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.”
See Glenn’s full EveryLife workshop presentation: https://everylifefoundation.org/event...
Request the Guide for Newly-Diagnosed Families: https://www.CureSFF.org/FamilyGuide
List of Foundation’s funded research grants: https://www.CureSFF.org/Grants
World Sanfilippo Awareness Day information: https://www.CureSFF.org/WSAD
Questions: Contact@CureSanfilippoFoundation.org
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Music credit: Springish by Gillicuddy
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