Cure Sanfilippo Foundation

To advocate for and fund research directed towards a cure or treatment options for patients with Sanfilippo Syndrome.

Every child is a precious gift. Every child deserves the opportunity to LIVE, without suffering and pain. Cure Sanfilippo Foundation is passionate about helping this one very basic wish for LIFE come true for children around the world and right here in Arizona.

Sanfilippo Syndrome (MPSIII) is a caused by a genetic enzyme deficiency that leads to a progressive dementia. The effects on a child's brain are similar to what is seen in Alzheimer's disease. Due to the rapid nature of the neurodegeneration, we are in a race against time. Symptoms begin to appear around 3 years of age, at a time when you are just beginning to know your little one's unique personality. Quickly though, the storage material in the brain builds up to a critical mass, causing loss of speech, sleeplessness, declining ability to learn and understand, seizures, and dementia. From there the child will need a feeding tube & wheelchair, and children commonly pass away in their teen years, yet this is preceded by these many years of suffering and disability. It is a devastating disease.

Today, no proven treatment or cure exists, but real HOPE is on the horizon. Breakthrough science has discovered treatment options which have proven in pre-clincial research models to slow or stop Sanfilippo. Cure Sanfilippo funds the most promising research to bring about more clinical trial options...a life line for these kids, before it is too late.

In 7+ years, thanks to incredible support and generosity, the Foundation has funded more than 30 research projects around the world, leading to first ever clinical trials in children. Some research areas funded include two gene therapy projects (now in clinical trial, and has treated 20+ children), enzyme-replacement, gene modified mesenchymal stem cell, high throughput screening, viral antibody suppression, among others. In essence, Cure Sanfilippo Foundation is funding research that is helping unlock mysteries of the brain, and could potentially bring insight into more common neurological disorders such as Alzheimer's and Parkinson's.

Every child is a precious gift. Every gift received during Arizona Gives, brings more HOPE to these families and children.

Testimonials

-- From Dr. Doug McCarty researcher formerly at Nationwide Children's Hospital: "Once we started working with the Sanfilippo Foundations, families and their children, we found it impossible to ignore the consequences of the (laboratory) experiments that we do. These scientific breakthroughs mean something very important to people & to children, and often they could be depending on this to survive. One of the roles of an advocacy group like Cure Sanfilippo Foundation, is that they provide funding, and that funding is critical…in addition they are challenging the scientists to continue searching for better results, and with every step we get better at what we are doing, and we get closer to a treatment or cure."

--- From Parents of Keira Esposito, (age 10, Sanfilippo Type A): "We know there are a lot of great causes and charities in the world worthy of funding. However, the rare disease community by definition is very small and we need all the help we can get. We make every dollar count. Please consider Cure Sanfilippo Foundation for your charitable giving, as they are helping to give Keira, and other children like her, a chance at life."

--- From Dr. Jeff Esko, Department of Cellular and Molecular Medicine at UC San Diego: "I have worked with Cure Sanfilippo Foundation for the last two years and watched the foundation grow in sophistication and reach. The work funded in my laboratory by the foundation allows us to examine a new delivery system for enzyme replacement therapy and potentially a novel route of administration. In these times of uncertain funding through the federal government, seed funding like this is paramount to finding new ways to treat MPS disorders (Sanfilipppo is MPS III). The foundation's portfolio is impressive and its impact cannot be underestimated."

--- From Dr. Jan Nolta, Director at Institute for Regenerative Cures at UC Davis School of Medicine: "The work that Cure Sanfilippo Foundation is doing to help children with MPSIII (Sanfilippo Syndrome) disease is incredibly important. They are completely devoted to helping scientists and physicians develop new cures for this disease that affects little children. In addition to fundraising to help the research move forward, they have worked tirelessly to raise awareness for children and families affected by this rare disease. Their Foundation's passion for the cause is unsurpassed."