The Foundation’s “A Close Look at Sanfilippo” podcast spends 10 minutes diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no FDA-approved treatment.
In podcast episode 12, president and co-founder of Cure Sanfilippo Foundation, talks about things happening in Sanfilippo today, things underway, and things to come.
(00:39) He kicks off with sharing the status of the next-generation gene therapy for Type A and B at University of North Carolina/Chapel Hill and projected timeline for clinical trials. He also announces that the Foundation is partnering with The Lundquist Institute for a new drug repurposing clinical trial that will be open to all types of Sanfilippo and likely children of all ages. He also shares the hopefully start date for this trial.
(02:00) O’Neill spends a moment talking about the Foundation’s most-recent grant funding round, where researchers globally were invited to submit their research ideas for funding. This year included a new fellowship award to stimulate early-career researchers’ interest in Sanfilippo Syndrome.
(02:46) O’Neill also touches on the upcoming Giving Tuesday and its importance relative to upcoming research projects. He shares the bold goal for this year’s Giving Tuesday, saying that “to better these children’s lives [those living with Sanfilippo Syndrome], we have to be aggressive and press the envelope.”
(03:41) He announces publication of the Foundation-led, first-ever consensus Global Clinical Care Guidelines for Sanfilippo Syndrome. The document contains extensive guidance on Sanfilippo Syndrome-specific care management and monitoring of disease-related changes, helping clinicians and families give children the best quality of life possible.
(04:59) He briefly revisits highlights from this summer’s Sanfilippo community conference, ADVANCE 2022, hosted by the Foundation, which are available as on-demand recordings.
(05:55) For the final topic of this podcast, he answers the most-common and hardest question that people ask him and his wife.
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.