Episode 11: “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 highlights from the 2022 WOLRD Symposium conference on lysosomal storage disorders. Including meetings with biotechs newly engaging in Sanfilippo research, as well as researcher Dr. Haiyan Fu of UNC Chapel Hill for an update on the Foundation-funded gene therapy research and when the clinical trial might begin (hint: likely this year). Glenn also shares his time spent with the Wacker family, whose daughter was recently diagnosed, and how they helped him out in a pinch.
Glenn talks about one of two poster presentations on CSF-funded projects at WORLD. The important poster presentation detailed early promising results from the Anakinra clinical trial. It reported that 82% of patients in the Anakinra trial had an improvement in at least one of the outcome measures after 8 weeks of treatment and 100% after 16 weeks.
Glenn then dives into important insights and considerations in relation to news that Abeona Therapeutics is closing its ABO-003 gene therapy clinical trial for Type A for patients with middle and advanced phases of Sanfilippo Syndrome. He addresses if this impacts whether gene therapy still holds a promise of benefit for children with Sanfilippo.
He closes this podcast episode sharing some of the amazing fundraising that have happened lately and are coming up and how the Foundation joined the rare disease community’s recent collective legislative advocacy to influence positive change for research on rare diseases.
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