Fighting for Time, Funding for Hope
At Team Sanfilippo Foundation, we recognize that time is everything for families affected by this genetic condition. With a mission to improve the quality of life of children diagnosed with Sanfilippo Syndrome, we support cutting-edge research for treatments and therapy. Our strategic plan is focused on accelerating research, funding the most promising science, and pushing hard toward effective treatments—and eventually, a cure.
Targeted Research Funding
We prioritize funding research that shows tangible potential to stop or slow the progression of Sanfilippo Syndrome, including gene therapies, enzyme replacement treatments, small-molecule drugs, and symptom management approaches.
Driving Collaboration
Collaboration helps speed up progress and avoid wasted time and resources. We actively build partnerships with doctors, scientists, biotech companies, and other advocacy organizations to share knowledge and maximize impact.
Targeted Research Funding
We prioritize funding research that shows tangible potential to stop or slow the progression of Sanfilippo Syndrome, including gene therapies, enzyme replacement treatments, small-molecule drugs, and symptom management approaches.
Driving Collaboration
Collaboration helps speed up progress and avoid wasted time and resources. We actively build partnerships with doctors, scientists, biotech companies, and other advocacy organizations to share knowledge and maximize impact.
Speeding Up Clinical Trials
We advocate for and support well-designed, accessible trials based on sound science. We also help families navigate trial enrollment and provide input to researchers to make trials more family-centered.
Listening to Families
A part of our strategy is guided by the voices of parents and caregivers. We believe families are experts in this disease, and we’re committed to supporting them, not just in the future, but also in the present.
Some children diagnosed with Sanfilippo Syndrome lose skills faster than treatments can reach them. Our strategic plan is not just a list of goals, it’s our roadmap to save lives.
Every dollar raised, every project funded, every partnership built is part of a bigger picture: giving families hope and children more time.
Our Research Priorities
Team Sanfilippo Foundation is focused on funding research that tackles the most pressing challenges of this disease:
Optimizing Emerging Treatments: We’re investing in research to maximize the effectiveness of promising therapies like gene therapy, ERT, and small-molecule GAG modifiers for children currently affected by Sanfilippo.
Targeting the Root of the Problem: We are studying and manipulating the genes altered in Sanfilippo that could unlock powerful new treatments. We’re exploring innovative approaches inspired by successes in other genetic disorders. We are finding which genes are up- and down-regulated as MPSIII progresses. We are investigating how switching on or off these genes provides therapeutic benefits, like in Angelman’s syndrome and topotecan.
Regenerating the Brain: We’re supporting research into stimulating the growth of new brain cells, which has shown promise in animal models of Sanfilippo. Our goal is to translate these findings into effective therapies.
Non-GAG accumulations in MPSIII: Our research goes beyond the primary GAG accumulation to investigate other harmful substances in the Sanfilippo brain. Aside from identifying and eliminating neurotoxins, we develop strategies to reduce them.
Lessons from Other Neuropathologies: We’re actively exploring how breakthroughs in treating other neurodegenerative conditions can be applied to develop effective therapies for Sanfilippo Syndrome.
