Sanfilippo Is a Rare Disease, But There Is Greater Awareness Than Ever.
The compassion of people is truly amazing. In five years, more than $8 million has been donated to the Foundation.
All of it coming from individuals who heard the stories of Sanfilippo families and made the selfless choice to help our kids have a chance at life. More than 40,000 strangers have stepped up to help find a cure for Sanfilippo Syndrome.
We work to keep elevating awareness by constantly telling the story. For one Sanfilippo family’s story is the reality for all of us.
Thought-Leadership Projects
Curing Sanfilippo is our ultimate goal, but your support also allows us to play a critical roll in influencing the road that leads there. Learn more about the Foundation’s thought-leadership projects.
Collaborations
Curing Sanfilippo is our ultimate goal, but your support also allows us to play a critical roll in influencing the road that leads there. Learn more about the Foundation’s collaborative mindset.
Sanfilippo Syndrome In The News
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 articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) as of September 2022....
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 articles and abstracts that are relevant to understanding, managing, and/or treating Sanfilippo Syndrome (mucopolysaccharidosis III or MPS III) as of August 2022....
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 described the use of an optimized gene therapy vector to treat a mouse model of Sanfilippo Syndrome Type B. Researchers used an AAV8 viral delivery vector that they had...
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 families of children with Sanfilippo Syndrome have been spreading awareness by sharing their journey with the disease and have amassed significant followings on TikTok over...
Natural History of Biomarkers & Clinical Outcomes | Type IIIA | Observational Study | Denali
Trial Information Denali Therapeutics is conducting a observational research study, which can also be called a natural history study, into Sanfilippo syndrome Type A (MPS IIIA). The purpose of the research study is to collect blood and behavioral information from...
Denali sponsoring observational research study (natural history) for Sanfilippo Type A
Denali Therapeutics has provided information about their observational research study natural history) for children with Sanfilippo Syndrome Type A. Below is a flyer provided by Denali. More information and sign-up is available here.From the study website, the purpose...