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
Research: Lysosomal storage disorders with altered heparan sulfate metabolism lead to dopamine-dependent autistic behaviors in Sanfilippo mouse models
A research project co-funded by Cure Sanfilippo Foundation and Sanfilippo Children's Foundation...
Family Friday Blog: The Merrick Family
Get to know the Merrick family – John, Lindsay, Caroline, and Georgia Kate! Find out what the past...
Foundation raises nearly $4 million with three GoFundMe campaigns to cure Sanfilippo Syndrome
Current campaign is nearing the $1 million mark to fund a first-ever clinical trial Cure...
Family Friday Blog: The Burroughs Family
Get to know the Burroughs family – Craig, Sara, Hannah, Addison, and Carson! Find out what the...
World’s first zebrafish model of Sanfilippo: A game-changer in Sanfilippo research
Dr. Jan Kaslin, from the Australian Regenerative Medicine Institute (ARMI), based at Monash...
Family Friday Blog: Loralei’s Family
Get to know Loralei and her family – mom Brandi, stepdad Cody, brothers Brayden and Blayne, and...
Family Friday Blog: The Rixer Family
Get to know the Rixer family – Andreas, Lisa, Saga, Casper, and Maia! Find out what the past year...
Free Guide: Tips to Help Kids Host a Successful Lemonade Stand
Lemonade stands are a hallmark of childhood summers Lemonade stands are a great opportunity for...
Family Friday Blog: The Willich Family
Get to know the Willich family – Jarrod, Megan, Beckett, and Kaisa!Find out what the past year has...
Family Friday Blog: The Shamaly Family
Get to know the Shamaly family – Kerry, Josephine, Alexander, and Ellie! Find out what the past...
Connor’s Crew: Children racing against time to save their best friend’s life
Most 12-year-olds are dreaming of what they want to be when they grow up, navigating school...
Family Friday Blog: The Bittner Family
Get to know the Bittner family – Josh, Lisa, Luke, and Caleb! Find out what the past year has been...
History being made: ScreenPlus, the largest U.S. newborn screening study, includes Sanfilippo
History is being made! Starting this week, newborns in the largest ever U.S. newborn screening...
Cure Sanfilippo supports North Carolina legislation HB 736 to expand state newborn screening
Cure Sanfilippo Foundation has joined other rare disease patient organizations in thanking the...
Family Friday Blog: The Karlson Family
Get to know the Karlson family – Matt, Deanna, Joseph, Addison, and Seth! Find out what the past...
Cure Sanfilippo joins in supporting expanded newborn screening legislation in Ohio
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to...
Cure Sanfilippo joins in calling Congress to support The BENEFIT Act
Cure Sanfilippo Foundation has signed-on to a stakeholder letter that calls for the U.S. Congress...
SOB-103 Enzyme Replacement | Type IIIA | Phase I-II | Sobi
Update: April 27, 2021 Last year Sobi decided to halt development of this ERT program. The...
EGT-101 Gene Therapy | Type IIIA | Phase I-II | Esteve
Update: April 2021 This Phase 1/2 trial has now fully enrolled and is not accepting additional...
Family Friday Blog: The Talbert Family
Get to know the Talbert family – Jeremy, Christina, Carson, Ryan, Lucas, and Parker! Find out what...
April 2021 Research Update: New research in 2021 and promising projects already underway
Children should have the chance to grow up. Parents should never have to watch their children...
Family Friday Blog: The Haywood Family
Get to know the Haywood family – Jason, Ashley, and Sadie! Find out what the past year has been...
Family Friday Blog: The Wallis Family
Get to know the Wallis family – Jeff, Kelly, Abby, and Emily! Find out what the past year has been...
Family Friday Blog: The Lagarde Family
Get to know the Lagarde family – Sam, Brittany, Sawyer, and Zion! Find out what the past year has...
Newborn screening pilot ScreenPlus that includes Sanfilippo Type A and B
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to...
Family Friday Blog: The Sarkar Family
Get to know the Sarkar family – Samir, Jen, Sophia, and Carter! Find out what the past year has...
Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo
Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation...
Family Friday Blog: The Hyder Family
Get to know the Hyder family – Jonathon, Nicole, Brayden, Gage, and Rylan! Find out what the past...
Family Friday Blog: The Byers Family
Get to know the Byers family – Tim, Valerie, Will, and Samantha! Find out what the past year has...
Free online CME course available on Sanfilippo syndrome
A free, online, continuing medical education (CME) course is available for any healthcare provider...
Family Friday Blog: The Esposito Family
Get to know the Esposito family – Dave, Elise, Kiera, and Keaton! Find out what the past year has...
New study: Economic burden of rare diseases estimated at $966B in 2019
In the most-comprehensive assessment of the total cost of rare diseases in the U.S. to date, the...
Family Friday Blog: The Smith Family
Have you met the Smith family – Nate, Alli, Spencer, and Sydney? Hear what the past year has been...
Family Friday Blog: Meet the Moon Family
Have you met the Moon family – Bill, Christine, Ella, Jacob, and Matthew? Hear what the past year...
Parents’ first viewing of a video they hope will save dying son
Connor Dobbyn is 12-years-old and has a degenerative and fatal disease called Sanfilippo syndrome....
Lysogene shares report on LYS-SAF302 biomarker data
Lysogene shared the following press release regarding its LYS-SAF302 Biomarker Data. The results...
Very special play list: Favorite songs of some children with Sanfilippo
Children with Sanfilippo syndrome love music! Up-tempo, toe-tapping songs that you can't resist...
WORLDSymposium 2021 is free for patients and families to attend; Cure Sanfilippo presenting
What is WORLDSymposium 2021 The WORLDSymposium 2021 conference, being held virtually Feb. 8-12,...
Nov. 16 officially recognized as Sanfilippo Awareness Day in Ohio
On Jan. 6, 2021, Ohio Governor Mike DeWine signed a bill that designates November 16 as Sanfilippo...
Thankful for you; Looking back at 2020 and ahead into 2021
Our goal at the beginning of the year was to raise $2 million in 2020, to help further research...
Investigating NDST1 inhibitors as target for substrate reduction therapy for Sanfilippo
Identifying a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that...
4 ways to make memories this holiday season
It's deep into the holiday season. And while this holiday season is unlike any others in our...
Merry & Bright Trivia Gala Night helps Foundation raise $2 million in 2020
We, at Cure Sanfilippo Foundation, are nearly speechless. $2 million in 2020 raised for research!...
AAV immunity is a significant issue for genetic therapies for all diseases
Glenn and Cara O'Neill, President and Chief Science Officer, respectively, of Cure Sanfilippo...
Peer-reviewed publication of first-ever Caregiver Preference Study on Sanfilippo
The first peer-reviewed publication of the Sanfilippo Caregiver Preference Study was released in...
Exploring anti-fungal immunity and the airway microbiome in Sanfilippo syndrome (MPS III)
Cure Sanfilippo Foundation is funding a two-year project by Neta Shlezinger, PhD, at the...
Families are the foundation of the Foundation
Every inch of progress towards finding a treatment and/or cure for Sanfilippo Syndrome happens...
A parent’s thoughts on a powerful day, World Sanfilippo Awareness Day
Today is a day that includes you You are more than just a donor to us. You are a cherished part of...
What We Thought Was Autism Became an Inconceivable Diagnosis
Connor Dobbyn was diagnosed with Autism at age 5. "We thought we hit the nail on the head with...
Save on your holiday cards and help cure Sanfilippo Syndrome
Now that it's November, people's thoughts are turning to the holidays. Early on people's holiday...
Identify a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that inhibit N-deacetylase/N-sulfo-transferase (NDST)
Cure Sanfilippo Foundation is joining Sanfilippo Children's Foundation (Australia) to co-fund a...
Foundation now accepting LOIs for Sanfilippo research funding
Cure Sanfilippo Foundation is now accepting Letters of Intent (LOI) for innovative research into...
Foundation’s Co-Founder & Chief Science Officer Cara O’Neill recognized as Rare Leader
Dr. Cara O'Neill, Co-Founder and Chief Science Officer of Cure Sanfilippo Foundation, was recently...
‘Save Connor’ viral video raises $100,000 in 27 hours for Sanfilippo clinical trail
FOR IMMEDIATE RELEASE MEDIA CONTACT: Glenn O’NeillPresident & Co-FounderCure Sanfilippo...
‘Save Connor’: Parents using viral video to overcome the single obstacle in the way of saving their son’s life: $3 million
FOR IMMEDIATE RELEASE MEDIA CONTACT: Glenn O’NeillPresident & Co-FounderCure Sanfilippo...
2nd annual World Sanfilippo Awareness Day to be celebrated Nov. 16, 2020
People around the world are making a significant difference for children with Sanfilippo Syndrome....
2020 Dino Dash 4 Declan 5K fundraiser enjoyed virtually
Even though the second annual Dino Dash 4 Declan wasn't able to happen as it did the year before,...
Foundation crosses $1 million mark in 2020 fundraising
Cure Sanfilippo Foundation supporters have raised more than $1 million in 2020 so far, to fund...
Medical records of Sanfilippo children can accelerate discovery of a treatment
Parents and caregivers of children with Sanfilippo Syndrome have the opportunity to help...
Lack of appropriate education for children with significant special needs
Dr. Cara O'NeillChief Science Officer for Cure Sanfilippo Foundation The pandemic has Cure...
Could your daily steps help save children’s lives? ????
You take thousands of steps every day. And those daily steps can help save children from...
Global Genes’ RARE Cast podcast interviews Cara O’Neill
Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, discussed her own journey...
“What is a life worth?” is an unconscious bias among clinicians
Dr. Cara O'Neill is Chief Science Officer for Cure Sanfilippo Foundation and mother to a...
A year after going public with son’s Sanfilippo diagnosis
A year ago, the parents of Connor Dobbyn decided to go public with his diagnosis of Sanfilippo...
‘Watch and wait’ approach to developmental delays is not acceptable anymore, says Dr. Cara O’Neill
Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, is also mother to a...
“I’ll do anything for my child” is pushed to the limits by Sanfilippo parents
Like every parent, Carrie and Caleb will do anything for their children. Which has led them to...
Support from fellow siblings of children with Sanfilippo and other disorders
Grey Chapin has carried a heavy load in her young life. Her older sister Blair died of Sanfilippo...
VIDEO: What Craig & Sara want to say to you
https://youtu.be/dOxewsJad4w This is Craig and Sara. Their beautiful son Carson is participating...
AmazonSmile now available in Amazon App
Shopping on Amazon = Saving Kids Today's reality of social distancing likely has you shopping...
Mom to face her greatest fear – skydiving – to save her daughter
https://www.facebook.com/CureSanfilippoFoundation/videos/283320393012600/ Haidyn's mom Carrie is...
Remembering the life of April Lucas
April Lucas passed away in June at the age of 50. She had Sanfilippo Syndrome Type C, one of the...
From our families to yours: Favorite recipes, perfect for summer
Here are more favorite recipes from our partner families, perfect for enjoying the summer season....
Fabric face masks now available in Foundation’s online store
Just like a cure can't wait for the pandemic, awareness won't either. Families and supporters are...
Cure Sanfilippo joins in calling for addition to Cures 2.0 legislation
Cure Sanfilippo Foundation is one of 46 patient advocacy organizations to sign-on to a letter...
Unique Clinical Trial Investigates a New Treatment Approach for Sanfilippo Syndrome
Trial Continues Despite COVID-19 Pandemic Investigators at The Lundquist Institute have teamed up...
The Big Give 2020 generates nearly $4,000 for Cure Sanfilippo
Thanks to many generous donors, the Foundation’s first-ever participation in The Big Give was a...
Foundation contributes to published list of early symptoms of MPS III
Cure Sanfilippo Foundation Chief Science Officer Cara O'Neill, MD, FAAP, and Board Member Valerie...
Cure Sanfilippo expands its website to provide additional information
Information, support, and resources regarding the more than 7,000 rare diseases in the world,...
Professional angler Mason Propst fundraising to cure Sanfilippo
"Not a day goes by that I don’t think about [my brother Hunter] or the disease known as Sanflippo...
Cure Sanfilippo participating in The Big Give 2020, June 11-12
The Big Give 2020 is a 25-hour online giving rally in Ohio to support nonprofits and equip them...
Key Points of Sanfilippo Advocacy Group Response to FDA Guidance
The combined Sanfilippo Advocacy group submitted comments to the U.S. Food and Drug...
Connor Beats Sanfilippo online campaign crosses $150,000 for Type C
In spring 2019, Connor Dobbyn's parents were told their son had Sanfilippo Syndrome, Type C (one...
Foundation Update | New Project & Progress – May 2020
This spring hasn’t been easy for anyone. But many generous people have made it a little less...
Creation of plasmid and efficacy study for MPS IIIC clinical trial
Cure Sanfilippo Foundation has funded New York-based Phoenix Nest, Inc. The funding supports...
Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy
Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS...
Devoted mom shaves her head for Cure Sanfilippo, raise awareness
On May 10, 2020 ... Mother's Day in the United States ... Jill Wood shaved off her lovely red hair...
“Light Up the Night” on May 15 to honor MPS Awareness Day
"LIGHT UP THE NIGHT" for MPS on Friday, May 15. Friday is a big day in the MPS community every...
Inspiring generosity on GivingTuesdayNow; $146,202 in donations
Uplifting Support Seen on GivingTuesdayNow Supporters around the world answered the call of "A...
Combined response to FDA draft guidance on drug development
On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group...
Sanfilippo caregivers invited to take RDCRN coronavirus survey
The novel coronavirus pandemic can have a serious impact on people with rare diseases and their...
A Cure Can’t Wait – GivingTuesdayNow
Life looks quite unfamiliar for all of us. But we take heart because one thing hasn’t changed:...
Survey: Caring For A Child With Rare Genetic Disorder During COVID-19
Families with a rare genetic disorder such as Sanfilippo Syndrome will have a different experience...
TIGEM identifies CLR01 as new potent drug candidate for MPS IIIA
Dr. Fraldi and his team at TIGEM (Telethon Institute of Genetics and Medicine) in Italy have been...
Foundation partner RDMD secures $14 million for expanded work
RDMD, which has partnered with Cure Sanfilippo Foundation and other select patient advocacy...
What does “better” mean and who gets to decide that?
Dr. Cara O'Neill, Chief Science Officer for Cure Sanfilippo Foundation, posed this question...
Coronavirus & Sanfilippo Syndrome: What Every Parent Should Know
How to Navigate Coronavirus As A Caregiver For A Child With Sanfilippo Syndrome The Coronavirus...
“George to the Rescue” show renovates Sanfilippo family’s home
"5-year-old Cameron Hyman suffers from Sanfilippo syndrome, a rare genetic condition. Six days...
Caregiver Preference Study for Sanfilippo presented to FDA
The urgent mission to save children from the fatal and rapidly-degenerative disease Sanfilippo...
Information on Coronavirus & people with chronic medical conditions
Parents of children with Sanfilippo Syndrome are already managing a complex medical condition,...
Video: Foundation provides scientific update, Coronavirus impact
Finding treatments and a cure for Sanfilippo Syndrome is the mission of the Foundation, it partner...