Podcast Episode 11: WORLD highlights, early Anakinra results, new gene therapy trial + more

March 28, 2022
Podcast episode 10 - Jan. 25, 2022

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 podcast episode 11, 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 (such at @Simons_Shot on TikTok) 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.

Podcast Episode 11’s Links

What is Sanfilippo Syndrome: https://curesanfilippofoundation.org/what-is-sanfilippo 

Symptoms of Sanfilippo: https://curesanfilippofoundation.org/symptoms 

Testing for Sanfilippo: https://curesanfilippofoundation.org/testing/ 

Past podcast episodes: https://curesanfilippofoundation.org/podcast/ 

Listen to the Podcast

Watch or listen to this episode of “A Close Look at Sanfilippo” at your convenience on Facebook, Instagram, or YouTube and all major podcast services, such as Apple Podcasts, Spotify, and Amazon Music.

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.

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