Current Clinical Trials & Studies for Sanfilippo

Page last updated: September 8, 2026

For web accessibility options: Click/tap the floating blue icon on the right.

Clinical trials and studies are where the rubber meets the road, scientifically.

Clinical trials for Sanfilippo are the crucial step of determining whether earlier research (basic and translational) shows the same therapeutic benefits in people.

Participation in observational studies and research surveys are opportunities to contribute to scientists’ understand of symptoms of Sanfilippo and how things change over time for the children. Participation also helps researchers develop new and improved outcome measures and more.

Current Clinical Studies in Sanfilippo Syndrome

DOWNLOAD: All Sanfilippo Clinical Trials At-A-Glance

Cure Sanfilippo Foundation has created a consolidated, printable, and easy-to-share document that details all current clinical trials for Sanfilippo Syndrome. Download it by clicking the button below. The information is for your reference and is not meant to replace consultation with your doctor or other sources. Information and changes to clinical trial offerings occur frequently and may not be reflected fully.

Contact us at Contact@CureSanfilippoFoundation.org if you would like more information about any of these clinical trial or other earlier-stage research underway.

We do our best to keep the list of clinical trials and studies for Sanfilippo up to date. However, we recommend periodically searching for “Sanfilippo” or “MPS III” on ClinicalTrials.gov and ClinicalTrialsRegister.eu in order for you to have the most-current information and to stay on top of newly-emerging clinical trial listings. Additionally, check back frequently with staff at Cure Sanfilippo Foundation and our website, as well as your medical team, for updates.

Individual Listings for Current Clinical Trials for Sanfilippo Syndrome

More Research on Sanfilippo

Beyond clinical trials, there is basic and translational research (earlier-stage research) exploring multiple therapies about how to address the systemic impact and symptoms of Sanfilippo in order to improve the quality of life for a child with Sanfilippo. Learn more about the entire ecosystem of Sanfilippo research, as well as current scientific news.

Research Funded by Cure Sanfilippo Foundation

Cure Sanfilippo Foundation architects and funds promising research to accelerate discovery of a treatment or cure for Sanfilippo. Explore the research funded by the Foundation.

Learn more about the Foundation

We are the nation’s largest 501(c)3 nonprofit dedicated to advocating and funding research to bring about treatments and/or a cure for Sanfilippo Syndrome. Cure Sanfilippo has funded research grants around the world which has helped clinical trials get underway, treating children with Sanfilippo Syndrome. The Foundation creates and explores innovative and breakthrough research to ensure multiple promising paths are being explored in the effort to save children and better their quality of life.

Parents of children with Sanfilippo meeting to contribute to the Foundation’s work to create materials to help scientists and regulators better include the patient and caregiver perspective in research.