Sanfilippo Syndrome and the fight to find a cure | Cure Sanfilippo Foundation

Imagine progressive dementia, but in children. That’s just part of what children with Sanfilippo Syndrome face.

This video explores:
What is Sanfilippo Syndrome, described by Dr. Cara O'Neill, a world-renowned scientific leader regarding Sanfilippo Syndrome | 00:10
A family's story about life with Sanfilippo Syndrome | 02:45
How Cure Sanfilippo Foundation is accelerating a first-ever treatment for Sanfilippo | 05:55

Sanfilippo Syndrome (also known as mucopolysaccharidosis III or MPS III) is a rapidly-degenerative and fatal disease that affects children of every gender, race, socio-economic level, nationality, and geographic area. These parents watch their young children lose all the skills they have gained in their short lives, suffer seizures and movement disorders, endure a lot of pain and suffering until they eventually die in their teens. There is currently no FDA-approved treatment or cure for Sanfilippo.

Cure Sanfilippo Foundation's mission: To develop and accelerate research in order to bring forward a first-generation therapy for children with Sanfilippo Syndrome.

Cure Sanfilippo Foundation is the largest U.S.-based, non-profit focused on finding a cure for these children and their families and is a highly sought-after collaborator globally. In the past two years, the Foundation has funded more than 12 research grants, including a clinical trial that treated 20 children and is showing promising results in early data. The outcomes of these, along with all 35+ research grants funded by the Foundation in its nine-year history, gives hope to every child with Sanfilippo today and for future generations because each is a step closer to a treatment or cure.

Every family of a child with Sanfilippo has a unique and heart-breaking story about the pain of living with this disease. Traumatized by the progressive loss of their child’s abilities, personality, voice,
and independence, these families learn to manage seizures, movement disorders, chaotic sleep, GI issues, and severe dementia. It consumes all of the family's resources and time, as they struggle to maintain their child’s worsening quality of life. They hope that, if not for their child, the future will have effective treatments available. Your support will help make that hope a reality.

Support this life-saving mission for children by making a donation at https://www.CureSanfilippoFoundation.org.

Learn more about Sanfilippo Syndrome:
What is Sanfilippo: http://curesanfilippofoundation.org/w...
Signs and symptoms of Sanfilippo: https://curesanfilippofoundation.org/...
Testing for Sanfilippo: https://curesanfilippofoundation.org/...
Research to cure Sanfilippo: https://curesanfilippofoundation.org/...

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