The following is a library of articles and information shared by the Foundation. Some are insightful and raw insights from our partner families about life with Sanfilippo, others are information about important research happening to find a cure for children with Sanfilippo Syndrome.
Questions?
If you have questions about these articles or anything else, we hope to hear from you. Reach out to us.
Articles & Posts
2026 Hands-on Workshop: “Does my insurance cover this?”
“Does my insurance cover this?” This is a common question Sanfilippo families face. If combing...
5 Things to Know | Sanfilippo Community Digest | Aug. 24, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things...
What Sanfilippo Families Need to Know: New Medicaid Work Requirements
Starting January 1, 2027, most states must require certain adult Medicaid enrollees to work,...
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB | MPS IIIB | Expanded Access | Spruce Bio
Page last updated: September 8, 2026 For web accessibility options: Click/tap the floating blue...
Erin Stoop, PharmD, mom of Olivia, joins Foundation Board of Directors
Erin Stoop, mom to 6-year-old Olivia (Sanfilippo Type B) is the newest addition to Cure Sanfilippo...
Natural History to Assess Disease in Patients With MPS IIIC (C-RARE) | MPS IIIC | Observational Study | UT Southwestern Children’s Medical Center
Page last updated: July 17, 2026 For web accessibility options: Click/tap the floating blue icon...
5 Things to Know | Sanfilippo Community Digest | July 10, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things...
Research platform for Sanfilippo syndrome, SanfilippoLINK, is now live and open for enrollment!
SanfilippoLINK is open to participants worldwide to advance understanding and treatments of all...
5 Things to Know | Sanfilippo Community Digest | June 12, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things...
Spruce shares long-term data from weekly TA-ERT for Sanfilippo Type B at international conference
This month, Spruce Biosciences shared long-term data from its tralesinidase alfa enzyme...
JCR to run clinical trial for JR-446 for Sanfilippo Type B in U.S. and Europe
Excited to see JCR Pharmaceuticals’ intravenous JR-446 enzyme replacement therapy (ERT) for...
Mother’s op-ed urges FDA to stay committed to rare diseases patients
"The FDA’s New Leader Must Keep the Agency’s Promises to Rare Disease Patients"Elise...
5 Things to Know | Sanfilippo Community Digest | May 16, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things...
Sanfilippo Type B AAV9 gene therapy program is picked up by biotech
Sangrail Biologics, a clinical-stage gene therapy company focused on rare pediatric genetic...
5 Things to Know | Sanfilippo Community Digest | May 4, 2026
“5 Things to Know” is a new, quick digest from Cure Sanfilippo Foundation of five things...
Study of Cannabidiol in Sanfilippo Syndrome | MPS IIIA, IIIB, IIIC, and IIID | Phase II-III | Lundquist
Page last updated: March 31, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility...
Foundation’s O’Neill among expert witnesses to testify before U.S. Senate Committee
On February 26, 2026, Cure Sanfilippo Foundation Chief Science Officer Cara O’Neill, MD, was among...
Leveraging clinical metadata in Sanfilippo syndrome: A scoping review, WORLDSymposium 2026 poster
This poster was presented at the WORLDSymposium 2026. Click on the poster to download a PDF of the...
Psychosocial burden in parents of individuals with Sanfilippo syndromes, WORLDSymposium 2026 poster
This poster was presented at the WORLDSymposium 2026 audience. Click on the poster to download a...
Ultragenyx resubmits UX111 to FDA for accelerated approval of Sanfilippo Type A
Ultragenyx announced today that it has officially resubmitted its application to the U.S. FDA...
January 2026 Sanfilippo Speak: Oral Motor Function Therapy for Sanfilippo Syndrome
How can you proactively strengthen your child’s ability to eat and drink by mouth? What are the...
Recent progress your generosity has helped make possible
An update on progress you made possible in 2025Your support has driven tremendous progress in...
Cure Sanfilippo participates in development, testing of Elsevier’s new AI tool LeapSpace
Cure Sanfilippo Foundation, the largest nonprofit in the U.S. dedicated exclusively to Sanfilippo...
Update: Sanfilippo Type B Expanded Access opportunity and fundraising status
Just a month and a half ago, an opportunity arose to support the creation of a Sanfilippo Type B...
November 2025 Sanfilippo Speak explores “total communication” with AAC
What is Alternative and Augmentative Communication (AAC)? What is “total communication”? How can...
Cure Sanfilippo Foundation awards 2025 grant to Greenwood Genetic Center
Photo courtesy of Greenwood Genetic CenterThe Greenwood Genetic Center (GGC) has been awarded a...
Opportunity for Expanded Access of ERT for some children with Sanfilippo Type B
The Sanfilippo community has been presented with an opportunity to have an Expanded Access enzyme...
October 2025 “Sanfilippo Speak” to focus on the ins and outs of estate planning
The next installment of "Sanfilippo Speak," Cure Sanfilippo Foundation’s family-focused webinar...
“FDA’s inaction blocks progress for a cure” says Foundation’s O’Neill in USA Today op-ed
"Right now, 30 million Americans – half of them children – have rare diseases, 95% of which have...
Neighbors, friends, and more make first Cure Clark 5K a huge success
More than 140 people turn out and help raise more than $10,000 to help children with Sanfilippo...
Foundation’s Dr. O’Neill publishes op-ed in The Hill, urging FDA to act
“The FDA must now ensure that its commitment to safety and efficacy is balanced with the urgency...
Ultragenyx provides additional information regarding its UX111 program for Sanfilippo Type A
Ultragenyx Pharmaceutical Inc. has provided additional information regarding its UX111 gene...
July 25 is Genetic Action Testing Day 2025
The inaugural Genetic Testing Action Day is July 25, empowering families to "Start Genetic" Cure...
FDA issues CRL for Ultragenyx’s UX111 gene therapy; company remains optimistic
On Friday, July 11, 2025, Ultragenyx Pharmaceutical announced that the U.S. Food and Drug...
First annual Mighty Maverick Muscle Run a huge success!
More than 200 people came out for the first annual Mighty Maverick Muscle Run in Lebanon, Ohio, on...
Family Friday: Our experience with hippotherapy and Sanfilippo Syndrome
The Clark family shares their experience with hippotherapy and Sanfilippo Syndrome Should parents...
Imagine Alzheimer’s and dementia in children. That’s Sanfilippo Syndrome.
Sanfilippo Syndrome is one of the most-devastating diagnoses a parent can receive for their...
Denali’s DNL126 trial for Sanfilippo Type A adds 6 more participants to cohort A3
Cure Sanfilippo Foundation is pleased to share that Denali Therapeutics has opened additional...
Recent signals regarding rare disease drug development from the FDA amidst changes
With many changes at hand in the federal government, the following are some insights on how the...
Spruce Bio announced acquisition of AX 250 ERT for Sanfilippo Type B
Spruce Biosciences has acquired Allievex’s tralesinidase alfa ERT (previously AX 250) for the...
Getting ready for school with the Stoop family – Life with Sanfilippo
Getting kids out the door to school is no easy task for any family. Add in Sanfilippo Syndrome and...
What to know about Hippotherapy and Sanfilippo, April 8 webinar
How can horseback riding integrate PT, OT, and speech therapy? What are its benefits for a child...
Family Friday: Tips for having a successful Make-A-Wish trip with Sanfilippo Syndrome
The Willich family shares their tips for having a successful Make-A-Wish trip with Sanfilippo...
Family Friday: Planning and managing long-distance travel with Asher
Walk through how the Bergeron family plans and manages long-distance travel to accommodate...
Family Friday: How the Doucette family keeps Emmett included in running errands
Come grocery shopping with the Doucette family of Saskatchewan, Canada! Find out how the Doucette...
Rare Disease Community Petition for Steady and Robust Federal Agency Leadership and more
EveryLife Foundation has created a petition on behalf of the rare disease community, asking for...
Cure Sanfilippo Foundation is now accepting Letters of Interest (LOIs) for research program
Open call for proposals to ADVANCE Sanfilippo syndrome researchCure Sanfilippo Foundation is now...
Cure Sanfilippo Foundation participating in WORLDSymposium 2025
Glenn and Cara O'Neill of Cure Sanfilippo Foundation are in San Diego, California, for...
Foundation’s Dr. O’Neill to speak at ASGCT’s Empowering Patients 2025 summit in March
Cure Sanfilippo Foundation Chief Science Officer and Co-Founder Cara O'Neill, MD, FAAP, will be a...
January 2025 “Sanfilippo Speak” to focus on ABA challenges and creative solutions
The next installment of "Sanfilippo Speak," Cure Sanfilippo Foundation’s family support webinar...
Foundation joins 200+ other organization, calling for long-term extension of PRV Program
Cure Sanfilippo Foundation, along with nearly 200 other organizations, was glad to be able to sign...
ADVANCE 2024 recordings available; watch sessions at your convenience
Recorded Sessions Available for On-Demand Replay The virtual ADVANCE 2024, a Sanfilippo Community...
Now recruiting: JLK-447 natural history study of Sanfilippo Type C
The natural history study of Sanfilippo syndrome Type C (study ID #JLK-447) sponsored by Phoenix...
Denali announces DNL126 demonstrates “robust reduction from baseline in CSF heparan sulfate levels, including normalization”
We are pleased to share that Denali Therapeutics has just announced positive preliminary...
Ambroxol Hydrochloride | MPS IIIA, IIIB, IIIC, IIID | Phase II-III | Lysosomal & Rare Disorders Research & Treatment Center
Page last updated: August 20, 2025 For web accessibility options: Click/tap the floating blue icon...
Action needed today: Senators need to hear from you
Good news and an urgent request to take action immediately … Two days ago, the Give Kids A Chance...
Opportunity for Action: Ask Congress to Support Creating Hope Reauthorization Act/PRV
The Rare Disease Pediatric Review Voucher (PRV) is set to expire in late September. This would not...
Further research confirms CLR01 reduces harmful clumping of abnormal proteins and brain inflammation, improves symptoms in Sanfilippo type A, B, and C mice
CLRO1 also enhances outcomes when combined with AAV gene therapyAssociate Professor Alessandro...
JR-446 Enzyme Replacement | MPS IIIB | Phase I-II | MEDIPAL/JCR
Page last updated: August 13, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility...
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC | MPS IIIC | Observational Study | Hospices Civils De Lyon
Page last updated: May 14, 2026 For web accessibility options: Click/tap the floating blue icon on...
Landmark ‘Nature Medicine’ Study Reports Promising New Treatment Reduces Suffering in Sanfilippo syndrome
In an open label study, the blood-brain barrier crossing anti-inflammatory anakinra was found to...
Effects of photobiomodulation in mouse model of Sanfilippo syndrome
Grant Details Project Title: Exploring novel photobiomodulation parameters in an animal model of...
What the Ultragenyx plans for Accelerated Approval filing announcement mean for Sanfilippo
On June 12, the milestone news was announced that Ultragenyx Pharmaceutical Inc. held a successful...
Breaking: FDA Agrees Heparan Sulfate is a Reasonable Surrogate Endpoint for UX111
MILESTONE BREAKING NEWS Ultragenyx Pharmaceutical Inc. announced just minutes ago that it held a...
Foundation launches family support webinar series, “Sanfilippo Speak”
Life with Sanfilippo Syndrome can be extremely isolating for parents and siblings. Few understand...
Denali’s DNL126 (MPS IIIA) program selected for FDA’s START pilot
Cure Sanfilippo Foundation is thrilled to see that a Sanfilippo program has been selected for...
GC1130A Enzyme Replacement | MPS IIIA | Phase I | GC Biopharma
Page last updated: July 29, 2025 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility...
Foundation-funded translational research identifies dopaminergic drug that improves autism symptoms and helps restore dopamine-receptor activity in the Sanfilippo A mouse model
The research project further investigated the impact of altered heparan sulfate (HS) metabolism on...
Record turnout for 2024 Super Eliza 5K; $15,000 raised
The 2024 Super Eliza 5K was an incredible day , raising more than $15,000 for research, bringing...
Foundation creates new position to elevate personalized, on-demand, Sanfilippo-specific support to families
Delivering the personalized information and support that you need, when you need itParents of...
“Saving Eliza” launched 10 years ago, eventually raising record-setting $2 million to cure Sanfilippo Syndrome
What do you do when you're told that your young, energetic, talkative child has a terminal illness...
Now available: Recording of Ultragenyx Type A Gene Therapy Program webinar
Watch recording of this week’s Webinar Update on Ultragenyx’s Sanfilippo Syndrome Type A Gene...
Evaluation of disease-modifying drugs for attenuated forms of Sanfilippo
Chief investigator Professor Kim Hemsley of Flinders University.The Sanfilippo Children’s...
Combination of stem cell transplantation and cathepsin B inhibitors for treatment of Sanfilippo
Chief investigator Alexey Pshezhetsky, PhD, of Centre Hospitalier Universitaire Sainte-Justine,...
“The Heart of Eureka” podcast speaks with Sanfilippo parents Jason & Theresa Wacker
Recently, The Heart of Eureka Podcast featured the Wackers, a local family whose daughter Gianna...
Qualifying Biomarkers to Support Rare Disease Regulatory Pathways: Focus on Neuropathic MPS
Page content provided by: Cure Sanfilippo Foundation, National MPS Society, and The Ryan...
March 26 webinar about Sanfilippo type A gene therapy program (UX111)
Ultragenyx is conducting a gene therapy clinical trial for Sanfilippo syndrome type A. This trial...
Research finds most-effective administration method for stem gene therapy in Sanfilippo Type B
A multi-year study, led by Coy Heldermon, MD, PhD, at University of Florida and co-funded by Cure...
Foundation is proud founding member of international Sanfilippo alliance ISSA
Today, we launch the International Sanfilippo Syndrome Alliance (ISSA), a global collaboration...
Substrate reduction candidate identified that can inhibit NDST1 enzyme activity
Sanfilippo Syndrome involves the accumulation of heparan sulfate sulphate in the body, resulting...
Special Books by Special Kids features another Sanfilippo family
Special Books by Special Kids (SBSK) is a nonprofit created by a former teacher of students with...
A Doctor And Mother’s Plea To FDA To Help Save Children With Rare Disease
The following opionion editorial by Dr. Cara O'Neill was published in International Business Times...
Generous donors help raise $1,850,000 in 2023 for Sanfilippo research
Thanks to YOU, more than $1.85 million was donated to Cure Sanfilippo Foundation in 2023 to help...
10 years ago, we never could have imagined …
This month celebrates 10 years since we established Cure Sanfilippo Foundation, after our daughter...
DNL126 Enzyme Replacement | MPS IIIA | Phase I-II | Denali
Page last updated: February 6, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility...
Denali ERT clinical trial, Sanfilippo Type A, UNC site also active
The below is a communication that Cure Sanfilippo Foundation received from Denali Therapeutics....
Denali ERT clinical trial, Sanfilippo Type A, UCSF site activated
Cure Sanfilippo Foundation is happy to share that we've been notified by the institution that a...
A commentary from Cure Sanfilippo Foundation about FDA’s Accelerated Approval pathway
The Washington Post published an article, "Her son is dying. She hopes the FDA will let her try to...
Giving Tuesday 2023 raises $324,753 for Sanfilippo research
Giving Tuesday 2023 fundraising total ... $324,753 raised! Families of children with Sanfilippo...
JR-441 Enzyme Replacement | MPS IIIA | Phase I-II | JCR
Page last updated: August 30, 2024 For web accessibility options: Click/tap the floating blue icon...
$8 million awarded to study brain cell death in fatal pediatric diseases, including Sanfilippo
Washington University School of Medicine (St. Louis, MO) has received nearly $8 million from the...
Families & friends fighting to ensure Sanfilippo research continues
We are so thankful for the many families who continue hosting events and fundraisers to support...
With more work, research, and clinical trials, we know [a therapy] is coming
Opening ADVANCE 2023, Glenn O'Neill, President & Co-Founder of Cure Sanfilippo Foundation,...
ADVANCE 2023 recordings; watch at your convenience
Recorded Sessions Available for On-Demand Replay The virtual ADVANCE 2023, a Sanfilippo Community...
Family Friday 2023: The Hooks Family
Hear directly from the Hooks family – Parents Cory and Jamie, Trenton (age 17, Type A), and...
Recent scientific articles relevant to Sanfilippo Syndrome: June – July 2023, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Family Friday 2023: The Nevins Family
Hear directly from the Nevins family – Parents Shannon and Jerad, Nathan (age 7, Type A), and...
Family Friday 2023: The Funderburk Family
Hear directly from the Funderburk family – Parents Josh and Jess, Jolene (age 5, Type A), and...
Foundation statement on video about Sanfilippo Syndrome circulating on social media
There are many compassionate and caring people who are just as eager as the families for there to...
Family Friday 2023: The Jamison Family
Hear directly from the Jamison family – Parents John and Courtney, Harper (age 4, Type A), and...