Page last updated: January 31, 2023 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Lysosomal and Rare Disorders Research and Treatment Center, Inc. is conducting a natural history study of patients with Sanfilippo...
Press Release
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 their daughter Olivia. Olivia has Sanfilippo Syndrome, a terminal degenerative disease in children that currently has no FDA-approved therapy or cure. But there is research...
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 U.S. Food and Drug Administration (FDA) and the Clinical Trials Transformation Initiative (CTTI) as a representative for the Patient Engagement Collaborative (PEC)....
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 articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) from October 2022 to...
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 icon on the right. Trial Information Phoenix Nest and National Institute of Neurological Disorders and Stroke (NINDS) are conducting a observational research study, which...
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 raise $1 million for research that can lead to a clinical trial that could save their 3-year-old daughter's life. On Saturday, Olivia's third birthday, they launched a...
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 Erin and Tyler should be spending today celebrating this precious milestone with cake and presents. Instead, they are launching a social campaign they hope will save...
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 topics regarding Sanfilippo Syndrome, a rapidly degenerative disease in children currently with no FDA-approved treatment. In podcast episode 12, president and co-founder of...
Now available: First-ever Clinical Care Guidelines for Sanfilippo Syndrome
For decades, clinicians and families have lacked clear guidance in making decisions about the timing and referral for medical monitoring and specialist care for children with Sanfilippo syndrome. Now the guess work is over.The first-ever global consensus clinical care...