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“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.
Episodes
Episode 12 – November 7, 2022
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about things happening in Sanfilippo today, things underway, and things to come. 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 the potential of the Foundation 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.
O’Neill talks about the Foundation’s most-recent grant funding round. He also announces publication of the Foundation-led, first-ever consensus Global Clinical Care Guidelines for Sanfilippo Syndrome. He briefly revisits highlights from this summer’s Sanfilippo community conference, ADVANCE 2022, hosted by the Foundation. For the final topic of this podcast, he answers the most-common and hardest question that people ask him and his wife.
Episode 11 – March 25, 2022
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 an update on the Foundation-funded gene therapy research and when the clinical trial might begin. Glenn also talks about the important poster presentation at WORLD, detailing early promising results from the Anakinra clinical trial. 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 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.
Episode 10 – Jan. 25, 2022
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about how the Foundation finished 2021 in fundraising and early generosity in 2022. Find out about the four key areas the Foundation, in collaboration Team Sanfilippo Foundation, will be addressing with the U.S. Food & Drug Administration (FDA) in a meeting to discuss better inclusion of the caregiver voice and preferences in research for Sanfilippo Syndrome. Glenn also shares details of two research grants that were recently approved by the Foundation’s board for funding. Hear how the Foundation is staying personally connected with its partner families, hear how they are doing, and get input on steering the Foundation. Glenn closed with explaining the significance of the upcoming WORLDSymposium conference and how the Foundation will be present. He shares excitement about two poster presentations regarding Foundation-supported projects, including one that will share results of the Anakinra clinical trial after 8 weeks of treatment.
Episode 9 – Dec. 18, 2021
In this episode of “A Close Look At Sanfilippo,” Dr. Cara O’Neill, Chief Science Officer and co-founder of Cure Sanfilippo Foundation, gives a year-end update on some important projects including the Anakinra clinical trial underway, funded fully by kind supporters of the Foundation. She goes on to talk about the UNC collaboration, other promising research, international collaborations, advocacy projects like addressing educational challenges and global clinical guidelines. She then talks about the Foundation’s focus in 2022. Glenn then talks about the Foundation history and impact from viral videos, and introduces the new Help Simon video launched.
Episode 8 – Nov. 15, 2021
In this episode of “A Close Look At Sanfilippo,” get the scoop on what’s happening globally in celebration of World Sanfilippo Awareness Day, the launch of a new viral video, why AllStripes could be a game-changer in the speed of research, who is invited to the Merry & Bright Trivia Gala Night, and what’s in store for GivingTuesday.
Episode 7 – Oct. 27, 2021
This episode explores 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 also introduces a new resource created for parents of children newly diagnosed with Sanfilippo, dives into how donors’ generous support of the Foundation has made more than 30 research projects possible in just eight years, and 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.
Episode 6 – Oct. 6, 2021
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about the inspiration and “why” behind creating a Foundation podcast; how the Step Up virtual steps challenge is shaping up for 2021 and how you can be a part of it; a recently co-funded research project at the University of Sidney to see if photobiomodulation, which has been explored as a therapy for several other neurodegenerative diseases, could provide multiple benefits in Sanfilippo; why so much money is needed for disease research; and the upcoming World Sanfilippo Awareness Day (Nov. 16) and it’s importance to the global community of families of children with Sanfilippo.
Episode 5 – Sept. 21, 2021
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about a recent presentation by Foundation Chief Science Officer Cara O’Neill, MD, FAAP, who offered multiple recommendations on how the “culture of the laboratory” can better integrate the true needs of patients, a facet of research that is sorely lacking. Additionally, Glenn talked about the kickoff of the 2021 Step Up to Cure Sanfilippo steps challenge and how people are using their steps to help children who have a disease that literally robs them of the ability to walk. He also addresses how newborn screening for Sanfilippo would be a “game changer” and the Foundation’s support of a newborn screening pilot in New York. Glenn also touches on the more than 100 partner families from around the world joining the Foundation and creation of a Professional Advisory Board to allow volunteers to lend their professional expertise to achieving the Foundation’s mission.
Episode 4 – Sept. 7, 2021
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about a free worksheet perfect for helping your child’s new teachers get to know them quickly so they build that critical rapport earlier; how the Pacl family has built a following of more than 1.5 MILLION followers on TikTok, sharing their son Logan’s life with Sanfilippo; why the Foundation completes the arduous process of an annual independent audit; how loyal supporters lift families’ spirits with their devotion and create a critical sustainable stream of income that enables the Foundation better plan what research it can fund next; and how we’re helping people celebrate the power of inclusion with a limited-edition collection of fun gear that also helps fund the mission to cure for Sanfilippo.
Episode 3 – Aug. 23, 2021
In this episode Glenn O’Neill, Foundation President and co-founder, shares a guide for educators and therapists about the important role they play in supporting neurocognitive function and quality of life in children with neurodegenerative diseases; Abeona Therapeutics’ encouraging news about its gene therapy; families globally seeking support and hope; and a resource uniquely for fathers of children with terminal illnesses. He is joined by Cara ONeill, Foundation Chief Science Officer and co-founder, to discuss publication of research about impacting a specific part of the dopamine pathway to reduce some Autism behavior symptoms.
Episode 2 – Aug. 5, 2021
In this episode Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, remembers and honors Reagan McGee; talks about how a new tool to test drugs that can lessen the buildup of heparan sulfate; gives a sneak peek the face of the Foundation’s next video campaign, coming out later this year; shares how the Foundation is walking the walk to ensure information accessibility; and details the Foundation’s latest grant round of more than $1 million in funding for five new research projects to help children with Sanfilippo.
Episode 1 – July 21, 2021
Glenn O’Neill, president and co-founder of Cure Sanfilippo Foundation, talks about the Foundation’s application for the Chan Zuckerberg Initiative Rare As One grant and how it could accelerate several global key research and collaboration projects; how use of biomarkers to approve a new Alzheimer’s drug could open doors for Sanfilippo and other degenerative diseases that can’t wait for “just perfect” options; how families of children with Sanfilippo sharing their medical records can accelerate scientists’ development of trials and treatments; the many ways people are fundraising and supporting the Foundation, enabling it to continue its mission to find a treatment or cure; and an update on the Anakinra clinical trial’s initial indicators and what comes next.
Thank you to the partner families, donors and supporters, researchers and clinicians, and industry partners that make all of the work and progress described in the “A Close Look At Sanfilippo”podcast possible.
Listen to the podcast on the Foundation’s YouTube channel.
Would love to hear your thoughts on this first episode and what you’d like to hear about in the next one. Reach out to Contact@CureSanfilippoFoundation.org.