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
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...
Family Friday 2023: The Nash Family
Hear directly from the Nash family – Parents Brandon and Ashley, Kinley (age 7, Type A), and...
Look what you made happen this weekend in Minneapolis!
On Saturday, you helped make cherished memories and build lifelong bonds of friendships. You’re...
Family Friday 2023: The Wallis Family
Hear directly from the Wallis family – Parents Jeff and Kelly, Abby (age 27, Type A), and sister...
Recent scientific articles relevant to Sanfilippo Syndrome: April – May 2023, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Family Friday 2023: The Kelly Family
Hear directly from the Kelly family – Parents Brian and Jen, Oliver (age 8, Type A), and sister...
Family Friday 2023: The Haywood Family
Hear directly from the Haywood family – Parents Ashley and Jason and Sadie (age 7, Type...
Recent scientific articles relevant to Sanfilippo Syndrome: January – March 2023, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Family Friday 2023: The Burroughs Family
Hear directly from the Burroughs family – Parents Sara and Craig, Carson (age 12, Type A), and his...
Jessica Haywood joins Cure Sanfilippo Foundation board
Jessica Haywood has joined the board for Cure Sanfilippo Foundation, bringing additional data...
Family Friday 2023: The Amos Family
Hear directly from the Amos family – Parents Kim and Ben, Liam (age 13, Type A) and Oliver (age 5,...
Family Friday 2023: The Sink Family
Hear directly from the Sink family – Parents Katharine and John, Declan, and his brother...
Family Friday 2023: The Barber Family
Hear directly from the Barber family – Parents Lauren and Sean, Autumn, and her brother Jack and...
Recording: Effect photobiomodulation in mouse model of Sanfilippo syndrome
Light therapy, also known as Photobiomodulation (PBM), has been explored and shown beneficial...
Family Friday 2023: The Chiara Family
Hear directly from the Chiara family – Parents Cecilia and Carlos, Maria, and her brothers Felipe...
Family Friday 2023: The Baxter/Muma Family
Hear directly from the Baxter/Muma family – Blaine, Brittany, Ashton, and Paislee!Awareness is a...
Family Friday 2023: The Stoop Family
Hear directly from the Stoop family – Tyler, Erin, Olivia, and Liam!Awareness is a huge part of...
Recognizing MPS Awareness Day 2023
Today, May 15, is MPS (Mucopolysaccharidosis) Awareness Day. Sanfilippo Syndrome's official name...
Family Friday 2023: The Esposito Family
Hear directly from the Esposito family – Dave, Elise, Keira, and Keaton!Awareness is a huge part...
Family Friday 2023: The Laughlin Family
Hear directly from the Laughlin family – Shea, Erin, and Spencer!Awareness is a huge part of...
U.S. patients: Denali’s natural history study still enrolling
Reminder: Denali Therapeutics is still enrolling patients with Sanfilippo Syndrome Type IIIA for...
Blood-based biomarkers for Sanfilippo syndrome progression and treatment
Grant Summary Institution: University of California, Los Angeles Primary Investigator: Gal Bitan,...
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC (MPSIIIC) | Type IIIC | Observational Study | Hospices Civils De Lyon
Page last updated: April 24, 2023 For web accessibility options: Click/tap the floating blue icon...
Tips for Make-A-Wish trips to Disney World, from a Sanfilippo family
Many families choose a trip to Disney as their Make-A-Wish. While exciting, a Make-A-Wish trip to...
O’Neill joins other rare disease advocates for meeting with FDA Commissioner, Directors
Last week, Cure Sanfilippo Foundation Chief Science Officer and Co-Founder Cara O'Neill was...
Family Friday 2023: The Ulrich Family
Hear directly from the Ulrich family – Jericho, Christin, Elise, and Veda!Awareness is a huge part...
2023 Super Eliza 5K brings annual event’s total to $100,000
Saturday, April 1, was the eighth annual Super Eliza 5K was extra special. This year, the event to...
$432,000 donated so far in 2023 to help children with Sanfilippo Syndrome
ANNOUNCEMENT The Foundation is thrilled to announce that, together, generous donors have helped...
Sanfilippo researchers, leaders discuss data sharing to advance Sanfilippo Syndrome therapeutic development
Meeting summary now available to download Sanfilippo syndrome is an ultra-rare, neurometabolic...
Denali announces plans to submit IND for ERT therapy DNL126 for Sanfilippo based on preclinical data
In a Feb. 22 press statement, Denali Therapeutics, based in San Francisco, California, announced...
Packed days for Foundation leaders attending WORLDSymposium 2023, including presenting
Page reviewed by: Dr. Cara O'Neill, FAAPPage last updated: March 14, 2023For web accessibility...
WILLPower team raises nearly $50,000 in 2023 The Woodlands Marathon Charity Challenge
It's been 8 years since Valerie and Tim Byers learned that their young son had Sanfilippo...
University of Manchester says gene therapy trial’s early results “show promise”
At WORLDSymposium 2023, Brian Bigger, Chair in Cell and Gene Therapy at University of...
2022 Annual Report: Look what you’ve done to help children with Sanfilippo
In late 2022, we shared with great excitement and appreciation that people's generosity was...
A Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) | Type IIIA, IIIB, IIIC, IIID | Observational Study | Lysosomal Center
Page last updated: January 31, 2023 For web accessibility options: Click/tap the floating blue...
Muskego Girls Basketball Association donates $33,409 from its 2023 “Swish for A Wish” to Saving Liv
The Milawukee-area community continues to rally around the Stoop family and its fight to save...
Foundation’s Cara O’Neill named to FDA and CTTI Patient Engagement Collaborative (PEC)
Dr. Cara O'Neill, the Foundation's co-founder and Chief Science Officer, has been selected by the...
Recent scientific articles relevant to Sanfilippo Syndrome: October-December 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
A Natural History Study of Sanfilippo Syndrome Type D (MPSIIID) | Type IIID | Observational Study | Phoenix Nest
Page last updated: December 13, 2022 For web accessibility options: Click/tap the floating blue...
Saving Liv video raises $100,000+ in first 36 hours; gathering media attention
Liv's parents give thanks for support of "Saving Liv" Olivia Stoop's parents are on a quest: To...
Saving Liv: Parents hope viral video will save their 3-year-old daughter from Sanfilippo Syndrome
[COLUMBIA, SC] - Today is Olivia “Liv” Stoop’s third birthday. Her “golden birthday.” Her parents...
Podcast Episode 12: New Sanfilippo drug repurposing clinical trial; updates on other research + more
The Foundation's "A Close Look at Sanfilippo" podcast spends 10 minutes diving into five hot...
Now available: First-ever Clinical Care Guidelines for Sanfilippo Syndrome
For decades, clinicians and families have lacked clear guidance in making decisions about the...
How rare disease awareness can be blended into Trick-or-Treat fun
Hand out a bit of awareness along with the Halloween candy this yearOne person at a time. Over and...
Recent scientific articles relevant to Sanfilippo Syndrome: September 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Recent scientific articles relevant to Sanfilippo Syndrome: August 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Testing of optimized gene therapy vector shows positive results in Sanfilippo type B mice
Newly-published research, led by Professor Coy Heldermon at the University of Florida, has...
Sanfilippo parents unite on TikTok to raise $1M in 1 month for research for the deadly childhood disease
TikTok community donates more than $20,000 in 24 hours to support Sanfilippo research Several...
Natural History of Biomarkers & Clinical Outcomes | Type IIIA | Observational Study | Denali
Trial Information Denali Therapeutics is conducting a observational research study, which can also...
Denali sponsoring observational research study (natural history) for Sanfilippo Type A
Denali Therapeutics has provided information about their observational research study natural...
Stories that give us hope: Ryan Dant and MPS I
The above story appeared earlier this week on The Today Show, which has helped raise awareness of...
Recent scientific articles relevant to Sanfilippo Syndrome: July 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Help Simon update: New video sharing two miracles; one more needed
When Alina and Jeremy started sharing Simon’s story and his fight with Sanfilippo Syndrome, they...
Researchers publish results of previously-terminated Sanfilippo Type A enzyme replacement study
Study results from Sobi's previously-terminated Sanfilippo Type A enzyme replacement study have...
Sanfilippo research funding available from Foundation
Cure Sanfilippo Foundation now have three funding opportunities available for innovative research...
Replay of ADVANCE 2022, Sanfilippo conference, available on-demand
The virtual ADVANCE 2022, Sanfilippo Community Conference, July 7-8, 2022, brought together...
Registration is open for ADVANCE 2022: Sanfilippo Community Conference
Registration is open for ADVANCE 2022: Sanfilippo Community Conference! A virtual conference on...
Recent scientific articles relevant to Sanfilippo Syndrome: June 9, 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Promising results from Lysogene MPS IIIA trial; novel PROVide study collecting observations
Lysogene announced May 18 that its AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302...
Caregivers define meaningful, relevant clinical outcome assessments for Sanfilippo Syndrome
Page reviewed by: Dr. Cara O'Neill, FAAP Page last updated: May 19, 2022 For web accessibility...
Sanfilippo Type A gene therapy ABO-102 program will continue, Ultragenyx acquires rights
Funding, endpoints, and more continue to make the road difficult in reaching an approved therapy...
A birthday message to Aidan: “I’ll love you until my last breath and never give up your fight.”
On May 10, Tina Holcomb shared the following words in honor of her son Aidan's 10th birthday in a...
“Strong As a Mother” – A mom’s poem about Sanfilippo Syndrome
This Mother's Day, Lisa Bittner put on a shirt that bore the message, "Strong Like a Mother." As a...
Recent scientific articles relevant to Sanfilippo Syndrome: May 4, 2022, digest
Every week thousands of scientific articles on various topics are published. Here are some recent...
Chive Charities hosts flash fundraiser for “Help Simon” and Cure Sanfilippo
On Tuesday, Chive Charities kicked of a first-of-its-kind fundraiser to help Simon and other...
“Our suffering will prove to be something bigger” shares Sanfilippo mom in podcast
Sara Burroughs, mom of Carson, who is 11 and has Sanfilippo Syndrome, was invited by Brian Roberts...
Would you believe TikTok is filled with kind-hearted people? This family has seen it.
Would you believe me that TikTok is a place filled with amazing, kind-hearted people? I know...
Podcast Episode 11: WORLD highlights, early Anakinra results, new gene therapy trial + more
The Foundation's "A Close Look at Sanfilippo" podcast spends 10 minutes (and a little more in this...
Community’s vision for Rare Disease Center for Excellence at FDA
The Speeding Therapy Access Today (STAT ACT), H.R. 1730/S. 670, includes creation of a Rare...
2.6M views: TikTok community rallies around ‘Help Simon’ parents hoping to save their little boy, who has amazing basketball skills
A TikTok video has inspired people to donate more than $63,000 in the past six days to the...
O’Neills recognized as Hometown Heroes; share their journey with Sanfilippo
Glenn and Cara O’Neill were recognized by 93.1 The Lake (Columbia, SC) in March as one of its...
Identifying Disease Modifiers in Drosophila Models of Sanfilippo (MPS IIIA)
Grant Summary Institution: Clemson University, South Carolina, U.S. Primary Investigator: Trudy...
Pace of transformative therapies to overwhelm newborn screening process; experts propose innovations
There has been explosive growth in the number of transformative therapies developed for diseases...
Discovery & validation of translational biomarkers for Sanfilippo childhood dementia
Page reviewed by: Dr. Cara O'Neill, FAAP and Jan Kaslin, PhD Page last updated: February 18, 2022...
Book recommendations: Talking to kids about differently-abled people
As a community of differently-abled people, it means so much when folks show love to our children...
Now available: A Global Roadmap For Sanfilippo Syndrome Therapies
Sanfilippo Children’s Foundation (Australia), Cure Sanfilippo Foundation (USA), and other...
Heparan sulfate accumulation identified as “key initiating event” in Alzheimer’s could benefit Sanfilippo research
Thirty years ago, Alan Snow, PhD, and Thomas Wight, PhD, hypothesized that heparan sulfate GAG...
Foundation lends support to Maryland and Mississippi newborn screening legislation
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to...
Podcast Episode 10: Kicking off 2022; upcoming key meeting with FDA; recent grants + more
The year's first (and episode 10) of the Foundation's "A Close Look at Sanfilippo" podcast spends...
How sharing medical records can advance new treatments | AllStripes partnership
Sharing your child’s journey with Sanfilippo can advance new treatments Because Sanfilippo...
Top 5 pages visited on Foundation’s website in 2021
Top-5 lists" are an interesting way to see what is at the forefront of people's minds. Here's a...
Podcast Episode 9: Anakinra Trial update; Collaboration with UNC; other research updates; + more
The Dec. 17, 2021, episode of the Foundation's "A Close Look at Sanfilippo" podcast spends 10...
“Doing nothing is the biggest risk in Sanfilippo”
Sanfilippo Syndrome is ideal for the FDA's Accelerated Approval Pathway. Families are willing to...
Research must begin with understanding a disease’s impact on the people who have it
At the 2021 WORLD Symposium on Lysosomal Diseases, Dr. Cara O’Neill presented the results of the...
Study explores psychological and physiological benefits of music therapy treatment for Sanfilippo
"Music therapy can be a useful form of treatment with multiple benefits for children with...
Help Simon video raises $50,000+ in first week; gathering media attention
"Help Simon," a viral videao aiming to raise $1 million for two promising research pathways for...
Foundation applauds North Carolina lawmakers for newborn screening legislation
Ealier this month, North Carolina Governor Roy Cooper signed critical legislation that updates the...
5-year-old boy’s phenomenal basketball potential will go unrealized unless a treatment is found for Sanfilippo Syndrome
Simon's family is using a viral video to fundraise for research that could lead to the first-ever...
Podcast Episode 8: BIG WEEK! World Sanfilippo Day & viral campaign; Trivia Gala; All Stripes; + more
The Nov. 15, 2021, episode of the Foundation's "A Close Look at Sanfilippo" podcast spends 10...
Podcast Episode 7: Ideal for FDA’s accelerated approval; guide for newly-diagnosed families; + more
The Oct. 27, 2021, episode of the Foundation's "A Close Look at Sanfilippo" podcast spends 10...
Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Sanfilippo
Grant Summary Institution: University of Naples Federico II, Naples, Italy Investigator: Luigi...
Exploring the role of heparan sulfate and dopamine as disease modifiers in Sanfilippo
Grant Summary Institution: Telethon Institute of Genetic Medicine (TIGEM), Pozzouli, Italy...
11-year-old Maine boy raises $3,000 for a Sanfilippo clinical trial to help a fellow Connor
Connor Ridley, an 11-year-old from Maine, is proof that people can make the world a better place,...