We Are Working To Cure Sanfilippo Syndrome.
Our mission is simple:
To advocate for and fund research directed towards a cure and treatment options for patients with Sanfilippo Syndrome.
About the Foundation
Explore how the Foundation began, who leads it, the families that partner with it, and the impact it has had since it was founded.
Cure Sanfilippo conducts and funds promising research, projects, and clinical trials. Learn more about our research efforts.
Together, a cure comes sooner. We collaborate with families, researchers, academic institutions, regulators, and bio-technology companies.
The more people who know about Sanfilippo, the more progress that happens to find a cure. So we help elevate awareness of Sanfilippo.
We set ambitious annual fundraising goals in order to fund research for every kind of Sanfilippo and to dictate and accelerate the pace of research.
Cure Sanfilippo connects families globally with the latest information on current research, as well as provides support for fundraising and caring for their children.
Cure Sanfilippo Foundation is a 501(c)3 Not-For-Profit Organization
All net funding goes to the urgent mission to advance treatment options to treat children with Sanfilippo.
This is led by proactive families of children with Sanfilippo Syndrome and their supporters, who work tirelessly to change the fate and future for these children.
Donations may be tax-deductible.
Latest Foundation News
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....
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....
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...
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...
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