News and information on issues relevant to you as a parent, caregiver, and advocate of a child with Sanfilippo Syndrome.
News From the Foundation
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 for Sanfilippo Syndrome (MPS III). Over the years, including recently, clinical trials for Sanfilippo Syndrome were discontinued for these many reasons. Which makes...
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 Facebook post. About how Sanfilippo Syndrome has impacted every aspect of their lives for the past 10 years. But also how they find the joy in the moments they have,...
“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 mom of a son with Sanfilippo Syndrome, Luke, it inspired her to write the following, which she shared on her family's Facebook page dedicated to Luke, "Hope Joy Love...
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 articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III)."Caregivers’ assessment of...
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 children with Sanfilippo Syndrome. Chive Charities, a 501(c)3 organization, traditionally supports the direct needs of underserved veterans, military families, first...
“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 to be the first guest on his inaugural BRob Inspired podcast, which is dedicated to sharing "ordinary people doing extraordinary things." Sara and Brian began the...
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 because we’ve experienced it. “Will you watch it with us?” With this six-word invitation, millions of people on TikTok opened their hearts and joined my husband Jeremy...
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 episode) diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no FDA-approved treatment. In podcast...
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 Disease Center for Excellence within the U.S. Food and Drug Administration (FDA). The legislation was introduced March 2021 and is currently assigned to the House Committee on...
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 couple’s GoFundMe account On Wednesday, March 16, Alina and Jeremy Croke shared their raw and emotional reaction to watching a video for the first time that they hoped...
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 “Hometown Heroes.” Glenn and Cara lead Cure Sanfilippo Foundation as President/Co-Founder and Chief Science Officer/Co-Founder, respectively. Part of the recognition included...
Identifying Disease Modifiers in Drosophila Models of Sanfilippo (MPS IIIA)
Grant Summary Institution: Clemson University, South Carolina, U.S. Primary Investigator: Trudy Mackay, PhD, Director, Clemson Center for Human Genetics and Self Family Endowed Chair of Human Genetics Duration: 24 months Start Date: January 2022 Type of Sanfilippo...
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 that must be provided to patients early in life. This makes the need for expanded and updated newborn screening nationwide, which has not kept pace with these medical...
Discovery & validation of translational biomarkers for Sanfilippo childhood dementia
Grant Summary Institution: Monash University, Victoria, Australia Primary Investigator: Jan Kaslin, PhD, Associate Professor at Australian Regenerative Medicine Institute Duration: 36 months Start Date: January 2022 Type of Sanfilippo studied: A Types of Sanfilippo...
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 through acceptance and inclusion. (I can speak personally when my daughter Eliza is shown this type of love.) Our children are often vocal, active, and spontaneous...
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 10 minutes (and a little more in this episode) diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no...
How sharing medical records can advance new treatments | AllStripes partnership
Sharing your child’s journey with Sanfilippo can advance new treatments Because Sanfilippo Syndrome is rare, researchers need more information directly from patients and their families to understand the condition. Your child’s progression, symptoms and health...
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 minutes (and a little more in this episode) diving into five hot topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no...
Foundation applauds North Carolina lawmakers for newborn screening legislation
Ealier this month, North Carolina Governor Roy Cooper signed critical legislation that updates the state's newborn screening program. Referred to as "RUSP alignment legislation," the new state law implements a three-year timeline in which the screening must begin for...
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 approved therapy for Sanfilippo Columbia, SC (Nov. 16, 2021) - Simon Croke from Austin, TX, has been sinking baskets on his Little Tykes hoop like a pint-sized pro since...