You are the key to the future.
Thank you for being the frontline to finding a treatment or cure for this disease.
We believe it will take a mix of complementary and/or supplementary treatments to truly come to a functional “cure” for Sanfilippo Syndrome. This may mean several different treatments over a child’s lifetime. That is the reason we fund extensive research in a variety of research areas.
Additionally, we spearhead and/or support initiatives that create valuable disease-specific and patient-preference information about Sanfilippo.
All of which leads to more effective treatments and more opportunities for earlier diagnosis.
Current Research on Sanfilippo Globally
Review the research avenues being investigated to find a treatment or cure for Sanfilippo Syndrome.
Research We Have FundedSee the list of research grants the Foundation has funded.
Research We Seek to FundCure Sanfilippo provides grant funding for promising research and projects with the following focuses:
- Pursuit of a treatment or cure for Sanfilippo Syndrome
- Expediting accurate diagnosis of Sanfilippo Syndrome
- Improved clinical design
- Improved integration of caregiver perspective and priorities in evaluating therapies
- Newborn screening of Sanfilippo Syndrome
- Rare-disease education
Interested in Support From the Foundation?
If you are interested in a grant from or collaborating with Cure Sanfilippo Foundation for a project regarding Sanfilippo Syndrome, please contact us via the form below. We look forward to hearing from you.
Resources for Researchers
Caregiver Preference Study for Sanfilippo Syndrome
Cure Sanfilippo Foundation is working to publish the first-ever Caregiver Preference Study for Sanfilippo Syndrome. It will include what parents consider “meaningful benefit,” as well as an exploratory staging tool.
Global Clinical Management Guidelines for Sanfilippo Syndrome
Cure Sanfilippo Foundation is leading the development the first-ever Global Clinical Guidelines for the management of Sanfilippo Syndrome, which are critical for patients and health care professionals in the management of rare diseases. The lack of experience and knowledge about a condition often causes late diagnosis and less than optimal management of the condition.
Analysis of the Caregiver Burden Associated with Sanfilippo Syndrome
A group of international clinical advisors with expertise in the care of pediatric patients with Sanfilippo and lysosomal storage disorders met to begin filling this void of understanding and create best-practice guidance for clinicians.
International Mice Registry
The International Mouse Strain Resource (IMSR) is a searchable online database of mouse strains, stocks, and mutant ES cell lines available worldwide, including inbred, mutant, and genetically engineered strains.
White Papers – Advancing Models of Patient Engagement: Patient Organizations As Research and Data Partners.
White Paper #1. “Making the Case” for partnership with patient organizations and the inclusion of patient-generated health data in the new era of data-driven research;
White Paper #2. “For Patient Organizations” aims to provide tools and guidance to help patient organizations improve their capacity as research and data partners; and
White Paper #3. “For Researchers” informs those conducting data-driven research about the specific capabilities patient organizations can bring and how to effectively partner with them.
FDA “Patients Matter” Video Series
The “Patients Matter” Video Series is a series of short videos developed by FDA’s Patient Affairs Staff to teach patients and other stakeholders about FDA and patient engagement efforts.