News and information on issues relevant to you as a parent, caregiver, and advocate of a child with Sanfilippo Syndrome.
News From the Foundation
Family Friday Blog: The Pearson Family
Get to know the Pearson family – Bruce, Cybill, Simone, Joana, and Solomon! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Pearson family's honest and insightful...
New enzyme assay testing method for Sanfilippo created with Foundation funding
A New and Improved Testing Option for Evaluating Potential Drugs for Sanfilippo In the fight to cure Sanfilippo syndrome, a key basic requirement is to be able to measure critical substances and to do so as accurately, easily, and inexpensively as possible. The study...
Cure Sanfilippo lends support to multiple provisions of Cures 2.0 Act
The 21st Century Cures 2.0 Act legislation from U.S. Representatives Diana DeGette (D-Colorado) and Fred Upton (R-Michigan) could have a profound impact on the speed of therapy development and diagnostic access for patients with rare diseases. "Many organizations have...
Family Friday Blog: The Sink Family
Get to know the Sink family – John, Katharine, Declan, and Zane!Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Sink family's honest and insightful perspective. The...
Family Friday Blog: The Koch Family
Get to know the Koch family – Matt, Julie, Alex, and Jaxson! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Koch family's honest and insightful perspective. The...
Cure Sanfilippo agrees: The need to use surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo
A powerful article from Dr. Emil D. Kakkis outlines the critical need for use of surrogate biomarkers in an accelerated drug approval pathway for diseases like Sanfilippo syndrome. In the article, "Aduhelm’s accelerated approval offers a promising roadmap for rare...
Family Friday Blog: The McKenzie Family
Get to know the McKenzie family – Kirk, Erica, Garrett, Reagan, and baby Ava on the way! Find out what the past year has been like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the McKenzies family's honest...
Save Connor campaign reaches $1 million for first-ever Sanfilippo Type C trial
The parents of Connor Dobbyn have shared a heartfelt message to supporters, announcing that the Save Connor campaign to fund the first-ever gene therapy clincial trial for Sanfilippo Syndrome Type C has reached $1 million. The announcement comes days after Connor...
Family Friday Blog: The Merrick Family
Get to know the Merrick family – John, Lindsay, Caroline, and Georgia Kate! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Merrick family's honest and insightful...
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 Sanfilippo Foundation is on track to raise another $1 million via a GoFundMe fundraiser for research and clinical trials to cure Sanfilippo Syndrome. This is the third...
Family Friday Blog: The Burroughs Family
Get to know the Burroughs family – Craig, Sara, Hannah, Addison, and Carson! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Burroughs family's honest and insightful...
Family Friday Blog: Loralei’s Family
Get to know Loralei and her family – mom Brandi, stepdad Cody, brothers Brayden and Blayne, and sister Sierra! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through their...
Family Friday Blog: The Rixer Family
Get to know the Rixer family – Andreas, Lisa, Saga, Casper, and Maia! Find out what the past year has been like in their lives with this week’s Family Friday.Get a glimpse into living with Sanfilippo Syndrome through the Rixer family's honest and insightful...
Family Friday Blog: The Willich Family
Get to know the Willich family – Jarrod, Megan, Beckett, and Kaisa!Find out what the past year has been like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the Willich family's honest and insightful...
Family Friday Blog: The Shamaly Family
Get to know the Shamaly family – Kerry, Josephine, Alexander, and Ellie! Find out what the past year has been like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the Shamaly family's honest and insightful...
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 cliques, and still enjoying the waning days of childhood innocence. This group of 12-year-olds have known Connor Dobbyn since kindergarten. Long before he was diagnosed with...
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 like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the Bittner family's honest and insightful perspective....
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 study, ScreenPlus, are being screened for Sanfilippo syndrome Type B! Addition of Sanfilippo Type A will follow in the coming months! Why invest in Newborn Screening...
Family Friday Blog: The Karlson Family
Get to know the Karlson family – Matt, Deanna, Joseph, Addison, and Seth! Find out what the past year has been like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the Karlson family's honest and insightful...
Family Friday Blog: The Talbert Family
Get to know the Talbert family – Jeremy, Christina, Carson, Ryan, Lucas, and Parker! Find out what the past year has been like in their lives with this week’s Family Friday. Get a glimpse into living with Sanfilippo Syndrome through the Talbert family's honest and...