Every parent dreams about their kid’s future and watching it unfold.
Sanfilippo Syndrome takes all that away, replacing it with pain and suffering. The child experiences severe dementia and dies in their mid-teens. All before their parents eyes.
Cure Sanfilippo Foundation architects and funds cutting-edge research to accelerate discovery of a treatment or cure for Sanfilippo Syndrome so parents can dream of their children’s futures again.
$12.5 million raised since 2013 15,000 children globally 35+ research projects funded 100+ partner families globally 3 clinical trials funded Medical & scientific expertise
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Every parent dreams about their child’s future and watching it unfold.
Sanfilippo Syndrome takes that all away.
Help save children
$12.5 million raised since 2013
Medical & scientific expertise
35+ research projects funded
Multiple clinical trials fundedDONATE
FUNDRAISE
TELL OTHERS
DONATE
FUNDRAISE
TELL OTHERS
By advocating for and funding research directed towards a cure and treatment options.
The Foundation’s focus in 2022: funding the most-promising research and elevating the voices of caregivers in pursuit of a cure for Sanfilippo syndrome.
Fundraising is critical fuel for these goals. Our goal is to raise $2,000,000 in 2022. We need your help to get there. Every single donation makes a difference.
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...
@curesanfilippo I believe. In honor of my daughter Eliza & all children with Sanfilippo ❤ #tiktokforacure #workyourmagic #curesanfilippo ♬ original sound - curesanfilippo