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
Connor Beats Sanfilippo online campaign crosses $150,000 for Type C
In spring 2019, Connor Dobbyn's parents were told their son had Sanfilippo Syndrome, Type C (one of the rarest types), and that it will take his life. Like Alzheimer's in children, Sanfilippo will cause Connor to lose all the skills he’s gained, stop speaking and...
Foundation Update | New Project & Progress – May 2020
This spring hasn’t been easy for anyone. But many generous people have made it a little less stressful for families of children with Sanfilippo Syndrome (MPS III). People's support of and generosity to Cure Sanfilippo Foundation have been the rainbow in a storm of...
Cure Sanfilippo funds $380,000 for first MPS IIIC gene therapy
Cure Sanfilippo Foundation funds $380,000 to Phoenix Nest Inc. to support path to first-ever MPS IIIC Gene Therapy COLUMBIA, SC (May 15, 2020) — Cure Sanfilippo Foundation is thrilled to announce funding of $380,000 to the New York-based biotechnology company Phoenix...
Devoted mom shaves her head for Cure Sanfilippo, raise awareness
On May 10, 2020 ... Mother's Day in the United States ... Jill Wood shaved off her lovely red hair to raise awareness and fundraise for research to cure children with Sanfilippo Syndrome, like her 13-year-old son Jonah....
“Light Up the Night” on May 15 to honor MPS Awareness Day
"LIGHT UP THE NIGHT" for MPS on Friday, May 15. Friday is a big day in the MPS community every year. It is International MPS Awareness Day. This Friday, May 15, when it gets dark, families, friends, and supporters will light candles outside their homes -- in their...
Inspiring generosity on GivingTuesdayNow; $146,202 in donations
Uplifting Support Seen on GivingTuesdayNow Supporters around the world answered the call of "A Cure Can't Wait" and donated an astonishing $146,202 on GivingTuesdayNow, May 5, to Cure Sanfilippo Foundation. Thirty-three families of children with Sanfilippo Syndrome...
Combined response to FDA draft guidance on drug development
On May 5, Cure Sanfilippo Foundation submitted the combined Sanfilippo Patient Advocacy Group Response to the U.S. Food And Drug Administration's (FDA) February draft guidance regarding the development of drugs to treat MPS III (Sanfilippo Syndrome). The 39-page...
Sanfilippo caregivers invited to take RDCRN coronavirus survey
The novel coronavirus pandemic can have a serious impact on people with rare diseases and their families. Yet, not much is known about it. The Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH), is conducting a...
A Cure Can’t Wait – GivingTuesdayNow
Life looks quite unfamiliar for all of us. But we take heart because one thing hasn’t changed: people are still thinking of others, finding ways to make a difference. Which is why families with Sanfilippo Syndrome are asking for help. Right now. Like everything else,...
Survey: Caring For A Child With Rare Genetic Disorder During COVID-19
Families with a rare genetic disorder such as Sanfilippo Syndrome will have a different experience than others during the COVID-19 emergency. Scientists at UCLA are working directly with families to better understand specific challenges and when they occur by...
TIGEM identifies CLR01 as new potent drug candidate for MPS IIIA
Dr. Fraldi and his team at TIGEM (Telethon Institute of Genetics and Medicine) in Italy have been working to identify a new treatment strategy for neurodegenerative lysosomal storage diseases. Cure Sanfilippo Foundation has supported this study thanks to the support...
Foundation partner RDMD secures $14 million for expanded work
RDMD, which has partnered with Cure Sanfilippo Foundation and other select patient advocacy groups, announced it has raised $14 million in Series A financing to continue its work to accelerate rare disease research and drug development. RDMD has developed an FDA-ready...