5 Things to Know | Sanfilippo Community Digest

“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things especially relevant to the Sanfilippo Syndrome community. We are going to send these regularly to keep you updated and put information and resources at your fingertips.

Most-Recent Edition

5 Things to Know | Oct. 14, 2026

1. ADVANCE 2026 recordings are now available!

The on-demand recordings of ADVANCE 2026 are now available. Hear the latest updates on clinical trials from industry leaders, exploration of emerging therapies and delivery platforms, and cast studies of real-world challenges our children face, such as episodes of unexplained distress, movement disorders, and participating in a clinical trial. This is a wonderful chance to hear detailed scientific explanations from the researchers and clinicians in the lab and working with Sanfilippo patients in the clinic.

Access the session recordings at: https://CureSanfilippoFoundation.org/ADVANCE2026.
*Translated captions in multiple languages can be activated on the recordings by clicking the YouTube [CC] icon.

Special thanks to the many families who shared their insight and experiences throughout the conference and provided planning and session feedback!

Thanks again to the ADVANCE 2026 sponsors – Denali Therapeutics, JCR Pharmaceuticals and MEDIPAL, Ultragenyx, Spruce Biosciences, Orchard Therapeutics, and GC Biopharma – for making this conference accessible for as many as possible, in part helping support this conference with live Spanish translations and registration for all attendees from around the world at no cost.

2. Maintaining momentum to bring forth more treatments, treatments for all

The approval of Fayuvi for Sanfilippo Type A last month is truly incredible and a “full circle” moment for the Foundation, having been one of our earliest funded programs. And while we celebrate, it represents only a single treatment option. So we can’t take our foot off the gas when it comes to research and the fundraising that fuels it.

We are continuing to fund the research and clinical trials that bring about additional treatment options — improved options, totally new therapies, treatments that work together to address the many challenges of Sanfilippo. For those still waiting for a treatment option. For those with Sanfilippo subtypes B, C, D. For those who aren’t eligible for Fayuvi. Even those who receive a treatment are likely to need advanced and combined treatments across their lifetime to address all the aspects of Sanfilippo.

You, the families, and your support networks have been at the heart of driving the fundraising for Cure Sanfilippo Foundation’s mission since day one, through to today. It’s incredibly inspiring and uplifting, and it’s what makes our community stand out! Many of you fundraise by hosting events or campaigns on social media to support the research happening right now for additional Sanfilippo treatments and the push for a cure. We are so thankful for all of you.

Here are just some of the amazing Cure Sanfilippo fundraiser events that have happened recently:

Fall Bake Sale, in honor of all children and families affected by Sanfilippo hosted by the Walton family in Ohio, on Oct. 10
12th annual Cure Sanfilippo Foundation Golf Classic, hosted by Eliza O’Neill’s family in Delaware, on Oct. 2
“Miles for His Smiles 5K,” hosted by Axel Steele’s family in Kentucky, on Sept. 20
2nd Annual Cure Clark 5K, hosted by Clark Willmore’s family in Michigan, on Sept. 26
Swing For A Cure Tournament, hosted in honor of Carson Burroughs and Gianna Wacker in Missouri, on Sept. 10
Wisco CrossFit Charity Golf Outing, hosted in honor of Olivia Stoop in Wisconsin, on Aug. 22
Birthday fundraiser celebrating Abby Wallis turning 31 years old, created by Abby’s family, on Aug. 8
2nd Annual Hope for Harper golf outings, hosted by Harper Jamison’s family, with Cure Sanfilippo being one of the beneficiaries
Donation from the 2026 Fat A** 5K & Street Party for Charity in Illinois, in honor of Lottie Millburn, in July
10th annual My Pal Spencer Golf Outing & Gala, hosted by Spencer Smith’s grandparents in New York, on July 20
2nd Annual Mighty Maverick 5K, hosted by Maverick Lana’s family in Ohio, on June 20

Current and upcoming fundraisers:

Rebel Clothing Collective: 10% sales through year end benefiting Cure Sanfilippo Foundation, thanks to Carter Sarkar’s family
4th Annual Love for Ashton “Shop for a Cure,” hosted by Ashton Baxter’s family in Washington, on Nov. 14
Giving Tuesday, hosted by Sanfilippo families everywhere, on Dec. 1. (This is historically our largest annual family fundraiser. Please join us, we will show you how and provide resources.)
“26.2 Miles for Frankie,” Houston Marathon, hosted by friends and family of Frankie Price, in Texas in January 2027

Have an idea for a fundraiser and want some support? Now is a great time to plan an event for early 2027! The Foundation has lots of experience and can help lighten the load of hosting a fundraising event, such as event registration, event contracts and deposits, signage, and more. Reach out to Foundation Senior Development Manager Lindsey Shealy at Lindsey@CureSanfilippoFoundation.org.

3. Next round of Aurora closed groups starting in November; Are you interested?

Life with Sanfilippo has highs and lows, laughter and grief, love and heartbreak. Sometimes all at once. You’re carrying multiple emotions that can be complicated, overwhelming, and difficult for most people to understand. You don’t have to navigate it alone. That’s what the Foundation’s Aurora Program is for.

The Aurora Program gives you a place to talk through what you’re experiencing, process the grief that comes with it, and find ways to care for yourself as you navigate life with Sanfilippo. All guided by our knowledgeable therapist with deep experience supporting Sanfilippo families and expertise in grief, traumatic stress, and resilience.

The next round of Aurora Closed Groups begins in November. In a “Closed Group,” you see the same parents every other week for six months, giving you the time and consistency to build relationships and create a space for honesty about what you’re experiencing, the good and the really difficult.

Interested in participating? Please complete the interest form by Sunday, Nov. 18.
https://forms.gle/Rks8ENPJYJXUoqF17

4. Struggling with out-of-pocket expenses due to Sanfilippo Type A?

A newly-available resource for financial support is available for the cost of FDA-approved treatment and other health-related services for Sanfilippo Type A from The Assistance Fund’s “IIIA-Sanfilippo A Syndrome Financial Assistance Program.”

Eligible individuals can receive assistance for a number of FDA-approved treatments for those with Sanfilippo Type A, including prescription drugs (copays, deductibles, and coinsurance), health insurance premiums, therapy administration costs, treatment-related travel costs, disease management (such as prescribing-physician copayments), and diagnostic laboratory tests, and genetic testing.

To learn more about The Assistance Fund or to apply today, visit https://enroll.tafcares.org/ or call (833) 426-2113. If you have questions about available financial assistance resources, contact Foundation Community Outreach and Education Coordinator Kassidie Reynolds at (803) 250-1985 or Kassidie@CureSanfilippoFoundation.org.

5. Meet the newest person working to help your children: Mohammed!

Over the past decade, increased awareness generated by Foundation campaigns, program funding, frequent speaking engagements, and collaborations has helped accelerate research and raise the level of recognition of Sanfilippo among researchers, companies, NIH, and FDA. Along with technology advances, these efforts have helped exponentially increase the pace of investigation and investment into Sanfilippo Syndrome.

Nothing is more urgent than continuing to accelerate research and therapies so treatments become realities for individuals of all ages and across the different forms of Sanfilippo. To continue this momentum, Cure Sanfilippo Foundation has expanded its science-focused staff. Join us in welcoming Mohammed Harris, PhD, MBA, MPH, MPA, as Research Programs Officer, Rare Disease Grants & Scientific Communications.

This role will directly impact the advancement of critical research programs and communications, helping the Foundation to continue shaping the rare disease treatment landscape, education and advocacy. Mohammed will oversee the Foundation’s extensive portfolio of multi-year funded research programs, lead the Foundation’s grant proposal process, and help digest and communicate scientific information to families and supporters.

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